Monthly Archives: July 2022

What Medicaid expansion means for new moms in Virginia – CBS 6 News Richmond WTVR

Posted: July 11, 2022 at 2:39 am

RICHMOND, Va. -- New moms receiving Medicaid assistance will now have their healthcare costs covered for a longer period of time.

Starting this month, Virginia Medicaid will expand from 60 days to 12 months of postpartum health coverage for enrollees.

Sara Cariano, a health policy analyst with Virginia Poverty Law Center, says the goal is to lower the maternal mortality rates in Virginia.

"A lot of women are still dying from pregnancy related complications after their coverage ends, so we want to get women in care, keep them in care, and not make them change health plans two months after having a baby," Cariano said.

This change applies those enrolled in FAMIS Moms and Medicaid for Pregnant Women.

"Medicaid and FAMIS cover a third of the births in Virginia, so this is really going to impact a lot of women, and a lot of these women previously, after 60 days, didnt have access to care because they didnt have coverage," Cariano said.

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According to the Virginia Department of Health, more than half of pregnancy-related deaths happen 43 days or more after the end of pregnancy. In Virginia, the mortality rate among Black mothers is more than two times higher than the mortality rate among white mothers.

Kenda Sutton-El, the Executive Director of Birth in Color RVA, works one-on-one with moms at higher risk.

"Theres always the stigma, especially when it comes to Black women, as soon as we go in there, the condition of our skin already puts us at a higher risk than other people," Sutton-El said. "One of the biggest concerns is that providers dont listen to what they have to say, or they dont feel comfortable telling their providers.

Non-English speakers and immigrants are also at higher risk of pregnancy-related health issues.

The folks who are the most vulnerable across the board who have the largest disparities are also most likely to not know about the coverage or a little nervous to enroll in it because they dont want it to interfere with immigration status or immigration proceedings," Cariano said.

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Dr. Tashima Lambert Giles, a board-certified OB/GYN with VCU Health, sees the impact of a lack of coverage first-hand.

"The truth is that a lot of our patients that are Medicaid have lower socioeconomic status. They have a lot more reasons to feel a lot more stressed, unsupported. They might have to get back to work a lot sooner than other moms," Dr. Lambert Giles said. "This might cause them to lose that access and lose the ability to recognize if theres something medically related thats going on, and not see a physician, because theyre continuing with normal life.

The expansion covers everything from regular check-ups, to substance abuse disorders, to postpartum depression care.

Dr. Lambert Giles said in her practice, she's seen more new mothers struggling with heart disease, underlying conditions left untreated, and mental health issues.

"I think Medicaid expansion allows us to tackle all of those things, but most importantly, getting preventative medicine to patients so that they overall patient is healthy, and we can get a community thats healthier," she said

Children born to Medicaid/FAMIS enrollees are entitled to 12 months of continuous coverage. Criteria and benefit details can be found here.

This is a developing story, so anyone with more information can email newstips@wtvr.com to send a tip.

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Healthcare for the Busy Bee – Flathead Beacon

Posted: July 11, 2022 at 2:39 am

After working as a registered nurse in hospital settings for several years, Lindsey Herndon became a board-certified Family Nurse Practitioner a few years ago, shifting toward a holistic practice to pair with western medicine.

Herndon worked in Oregon for the past few years at a family practice where she embraced preventative healthcare, which is where she came up with the idea to open her own mobile business to offer both telehealth and mobile visits to patients homes.

Teaming up with local nurses and a business professional, Herndon launched BusyBee IV Hydration and Wellness, a mobile healthcare company offering traditional services akin to an urgent care, intravenous (IV) therapies, vitamin shots and Botox.

Instead of working more towards fixing the problem as it happens, weve shifted toward preventative healthcare so with the idea of IV, hydration and mobile urgent care, were taking that holistic approach to bring to the patient in the comfort of their own home, Herndon said.

With services treating a variety of ailments, ranging from respiratory infections to skincare to hangover relief, Herndon and her business partner, Megan Scameheorn, and her crew of registered nurses, Liz Gidley, Amy Bottomley and Meghan Neufeld, also offer group packages.

The Hangover Helper IV drip has become one of the most popular therapies, providing 1,000 ML of fluid, B complexes, magnesium, manganese, copper and selenium.

Customers often book IV therapy parties for events like birthdays, bachelor and bachelorette parties as a group activity before or after long weekends.

Its a fun experience for everybody, Scameheorn said We had a client who booked us for a birthday party, and she wanted to make sure everybody got hydrated when they went back to work and got their hangover cure.

In addition to hangover relief, another popular IV drip is the Worker Bee, which is geared toward athletes recovering from strenuous training and contains fluids and a variety of vitamins and minerals. The therapy is designed to address muscle soreness, fatigue and enhance athletic performances.

Other IV therapies include the Myers Cocktail, the Thirsty Bee and the Beeautiful, each containing fluids, vitamins and minerals geared toward general wellness, hydration and skincare.

Herndon and Scameheorn are working with event organizers to potentially set up a booth at the Whitefish Marathon and The Last Best Ride.

In addition to IV drips, Busy Bee offers vitamin shots containing B vitamins, Vitamin D, antioxidants, biotin and a Skinny Shot that helps reduce sugar cravings.

This is a town where people work hard and play hard and they need to feel good in order to operate well, Herndon said. So we have a holistic approach of doing urgent care, IV hydration and even aesthetics We treat the individual from the inside out so they can function and do the things they love to do.

For more traditional services, Busy Bee offers telehealth and in-home treatment for gastrointestinal issues, respiratory infections, urinary tract infections, COVID testing and earwax removal.

I think its really exciting to have the opportunity to promote wellness in our community outside of the hospital setting, Neufeld said.

For more information, visit http://www.busybee-iv.com.

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The COVID BA.4 and BA.5 subvariants are highly transmissible. Here’s what else people in CT need to know. – CT Insider

Posted: July 11, 2022 at 2:39 am

Connecticut has seen an upward trend in COVID-19 cases over the past few weeks. While it may be possible that this increase is due to the new Omicron subvariants BA.4 and BA.5, public health officials say that its too soon to tell.

Dr. Manisha Juthani, commissioner of Connecticuts public health department, said its unclear exactly how widely BA.4 and BA.5 are spreading in Connecticut or the degree to which the new subvariants are contributing to the states recent uptick in COVID-19 cases and test positivity rate.

Its hard to say that the slight rise that were seeing is related to (the subvariants) or not, she said. Are we seeing this rise because of that? It is possible. I just dont have enough conclusive evidence to say that its absolutely the reason why.

Juthani, who was previously an infectious disease specialist at Yale New Haven Health, said that while the states COVID-19 numbers are still lower than they were this spring, the upward trend is increasingly hard to deny.

I dont want to sound the alarm every few weeks and then people start tuning out any sort of alarms, she said. But what I can say is that we are going in that direction.

Connecticut, like every other U.S. state, does not determine the variant of each positive case of coronavirus. Instead, the state Department of Public Halth, and Centers for Disease Control and Prevention, work with scientists, universities, hospitals and diagnostic labs to collect representative samples of positive tests for variant determination. These results are used to generate prevalence models to help public health officials estimate which variants predominate in states and across regions.

Yale New Havens Clinical Virology Lab, which mostly tracks outpatient samples from Fairfield, New Haven and New London counties, indicates that new cases are being driven by variants BA.2, BA.4 and BA.5. Regionally, the CDC estimates that the majority (roughly 42%) of new cases in New England are caused by BA.5.

According to Connecticuts public health department, new cases are up statewide with higher concentrations in the high population corridor between Stamford, New Haven and Hartford. This is despite overall higher vaccination rates in those areas.

Hospital utilization is up in the same area according to the federal Department of Health and Human Services. This upward trend can be seen across New England and New York. Every New England state but Vermont has over 70% hospital utilization. Connecticut is in the middle portion of the pack at 75% utilization. Rhode Island leads at 91%.

BA.5 has been reported to be more transmissible and immune evasive than previous strains of the coronavirus. Several studies have demonstrated that both BA.4 and BA.5 are more able to escape antibodies than previous variants. This is true for monoclonal antibody treatments, antibodies from prior infection and antibodies from vaccination.

Because of this immune evasion reports are emerging of more rapid reinfections than before. A CDC study documented ten cases of reinfection within 90 days of prior infection with the Delta variant across four states.

There have been a series of variants over time that have shared a couple characteristics, said Mark Adams deputy director at The Jackson Laboratory for Genomic Medicine. One is increased transmissibility ... but increasingly they seem to be driven by the ability to escape prior immune protection from SARS-COV-2.

The upshot is that even though this variant is more infectious it is not clear that it causes more severe or unusual infections. A large-scale study from Qatar indicates that vaccination is still extremely (97%) effective at preventing the worst outcomes even if vaccinated people still get sick.

It can infect people who have been previously infected but they tend to get a very mild infection, said Dr. William Schaffner, a professor of preventative medicine at Vanderbilt University Medical Center.

While some reports have emerged about unusual symptoms, such as viral meningitis, its not clear that this is a function of the new strain or a function of more cases increasing the likelihood of documenting rare complications.

We have heard, anecdotally, stories of a variety of symptoms (and recurrent infections) said Schaffner. But it isnt clear if its long symptoms or relapse or recurrent infection.

Its also not clear yet whether this wave of BA.4/5 will cause hospital capacity issues locally like it did in Portugal earlier in the spring. Transmission, severe infection and hospital use are complicated to predict.

Its really hard to predict the number of infections and the number of hospitalizations, said Adams. He encouraged people to get boosted if they could.

Its a real benefit. All the studies show that vaccination and boosting reduce the severity of disease, said Adams. The difference might be smaller (with the new variants) but its not going to be nothing.

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The COVID BA.4 and BA.5 subvariants are highly transmissible. Here's what else people in CT need to know. - CT Insider

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NHS to test using drones to fly chemotherapy drugs to Isle of Wight – The Guardian

Posted: July 11, 2022 at 2:39 am

The NHS plans to use drones to fly chemotherapy drugs to cancer patients in England to avoid the need for long journeys to collect them.

The devices will transport doses from Portsmouth to the Isle of Wight in a trial that, if successful, will lead to drones being used for similar drops elsewhere.

They will take 30 minutes to travel across the Solent, which will save patients on the island a three to four-hour round trip by ferry or hovercraft.

On Tuesday, Amanda Pritchard, NHS Englands chief executive, unveiled the move to help mark the 74th anniversary of the health services creation by the postwar Labour government.

Delivering chemo by drone is another extraordinary development for cancer patients and shows how the NHS will stop at nothing to ensure people get the treatment they need as promptly as possible, while also cutting costs and carbon emissions, she said.

The first drone deliveries will start shortly, NHS England said, subject to the outcome of the last of a series of test flights on Tuesday.

It plans to use the drones electrical vertical takeoff and landing aircraft to collect the medications from the Queen Alexandra hospital in Portsmouth and fly them to St Marys hospital on the Isle of Wight, where staff will collect and distribute them.

The drones weigh 85kg, have a wingspan of 5 metres and can carry up to 20kg. The scheme is the result of a partnership between NHS England and the technology company Apian.

This project marks a very important first step in the construction of a network of drone corridors connecting hospitals, pathology labs, GP surgeries, care homes and pharmacies up and down the country, said Alexander Trewby, Apians chief executive.

If the flights prove successful it will be much more convenient for the majority of cancer patients on the Isle of Wight who now have to travel to the mainland to receive their drugs.

Darren Cattell, the chief executive of the Isle of Wight NHS trust, stressed that we are still at a relatively early stage of drone use in healthcare but that drone could have radical and positive implications for both the NHS and for patients across the UK as well as the Isle of Wight.

Sajid Javid, the health secretary, said: I want England to become a world leader in cancer care and using the latest technology to deliver chemo by drone means patients will have quicker, fairer access to treatment no matter where they live.

Meanwhile, a study has found that reinviting patients every year to be screened for bowel cancer the UKs second biggest cancer killer could speed up diagnosis and save lives.

Although the proportion of people taking up the NHSs invitation to get screened has risen to 67%, bowel cancer has the lowest participation rate of all the health services screening programmes.

New research by Sheffield University showed that sending people a new home testing kit every year until they return one could prompt 13.6% more people to do so.

The study was funded by Cancer Research UK (CRUK) and is published in the journal Preventative Medicine.

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Michelle Mitchell, CRUKs chief executive, said: Screening is an effective way of catching cancer early and saving lives, but not everyone engages equally, and this contributes to health inequalities across the UK.

This study shows that sending yearly test kits to those who dont complete them could help close this gap and save lives.

The test used, the faecal immunochemical test, better known as the FIT test, looks for traces of blood in someones faeces. At the moment everyone in England aged 60-74 who is registered with a GP is sent one every two years. However, the government has pledged to expand the programme to 50- to 59-year-olds and the NHS has begun inviting 56 and 58-year-olds for screening.

Genevieve Edwards, the chief executive of Bowel Cancer UK, said: We know that once someone has taken part in bowel cancer screening, theyre more likely to do so again. So it will also be vital to increase investment in endoscopy and pathology staff and equipment, to match an increase in demand for prompt follow-up tests.

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The Role of Smoking and Body Mass Index in Mortality Risk Assessment for Geriatric Hip Fracture Patients – Cureus

Posted: July 11, 2022 at 2:39 am

Background

Smoking, obesity, and being below a healthy body weight are known to increase all-cause mortality rates and are considered modifiable risk factors. The purpose of this study is to assess whether adding these risk factors to a validated geriatric inpatient mortality risk tool will improve the predictive capacity for hip fracture patients. We hypothesize that the predictive capacity of the Score for Trauma Triage in the Geriatric and Middle-Aged (STTGMA) tool will improve.

Between October 2014 and August 2021, 2,421 patients >55-years-old treated for hip fractures caused by low-energy mechanisms were analyzed for demographics, injury details, hospital quality measures, and mortality. Smoking status was recorded as a current every-day smoker, former smoker, or never smoker.Smokers (current and former) were compared to non-smokers (never smokers).Body mass index (BMI) was defined as underweight (<18.5 kg/m2), healthy weight (18.5-24.9 kg/m2), overweight (25.0-24.9 kg/m2), or obese (>30 kg/m2). The baseline STTGMA tool for hip fractures (STTGMAHIP_FX_SCORE) was modified to include patients BMI and smoking status (STTGMA_MODIFIABLE), and new mortality risk scores were calculated. Each models predictive ability was compared using DeLongs test by analyzing the area under the receiver operating curves (AUROCs). Comparative analyses were conducted on each risk quartile.

A comparison of smokers versus non-smokers demonstrated that smokers experienced higher rates of inpatient (p = 0.025) and 30-day (p = 0.048) mortality, myocardial infarction (p < 0.01), acute respiratory failure (p < 0.01), and a longer length of stay (p = 0.014). Comparison among BMI cohorts demonstrated that underweight patients experienced higher rates of pneumonia (p = 0.033), decubitus ulcers (p = 0.046), and the need for an intensive care unit (ICU) (p < 0.01). AUROC comparison demonstrated that STTGMA_MODIFIABLEsignificantlyimproved the predictive capacity for inpatient mortality compared to STTGMAHIP_FX_SCORE(0.792 vs. 0.672, p = 0.0445). Quartile stratification demonstrated the highest risk cohort had a longer length of stay (p < 0.01), higher rates of inpatient (p < 0.01) and 30-day mortality (p < 0.01), and need for an ICU (p < 0.01) compared to the minimal risk cohort. Patients in the lowest risk quartile were most likely to be discharged home (p < 0.01).

Smoking, obesity, and being below a healthy body weight increase the risk of perioperative complications and poor outcomes. Including smoking and BMI improves the STTGMAHIP_FX_SCOREtool to predict mortality and risk stratify patient outcomes. Because smoking, obesity, and being below a healthy body weight are modifiable patient factors, providers can counsel patients and implement lifestyle changes to potentially decrease their risk of longer-term poor outcomes, especially in the setting of another fracture.For patients who are former smokers, providers can use this information to encourage continued restraint and healthy choices.

The worldwide population is aging. The World Health Organization (WHO) predicts that by 2030, one in six people will be 60 years old or older [1]. This trend toward an older population carries with it a higher risk of falls or accidents with subsequent orthopedic injuries. For example, the 2016 National Trauma Database found that patients older than 55 years of age comprised 42.6% of overall trauma and 57.6% of the deaths associated with these traumas [2]. Hip fractures, in particular, carry high rates of morbidity and mortality in the geriatric population [3]. Associated factors for poor outcomes in these patients include age, male gender, the presence of comorbidities, delayed time to surgery, and baseline ambulatory status [4,5]. As age, and to a certain degree, comorbidities are non-modifiable risk factors, it is important to consider factors that can be modified to lower a patients risk.

Body mass index (BMI) and smoking status are two such modifiable risk factors. Literature regarding the association of BMI and mortality or morbidity risk following hip fracture is divided. Despite an apparent obesity paradox, with obese patients having a lower risk of mortality, several studies have found contrasting results where obese, super-obese, and very underweight patients have higher rates of poor outcomes and mortality [6-9]. Similar to BMI, current research has demonstrated smoking to be associated with worse perioperative outcomes and higher rates of mortality following surgery [10-13]. Tobacco smoking is the leading cause of premature mortality that can be adjusted through behavioral changes, regardless of tobacco amount, as Qin et al. reported that even light smoking, that is, one to two cigarettes a day, can increase a patients all-cause mortality [14-16]. In former smokers, understanding the increased mortality risks is important to be able to provide preventative medicine and help these patients to remain smoke-free.

As hip fractures carry a significantly high rate of morbidity and mortality at baseline, it is important to consider strategies to decrease a patients mortality risk. Addressing and understanding modifiable risk factors is one way providers can intervene to improve outcomes. The Score for Trauma Triage in the Geriatric and Middle-Aged (STTGMA) is a validated inpatient mortality risk assessment tool for middle-aged and geriatric patients 55 and older who sustain different orthopedic trauma injuries [17]. The original STTGMA tool utilized clinical data available at the time of arrival to the emergency department (ED) to calculate a mortality risk score. Variables included in the original STTGMA tool were a patients age, injury details, Glasgow Coma Scale (GCS) score, and comorbidity profile as defined by the Charlson Comorbidity Index (CCI) [17]. Since STTGMAs inception, the model has evolved to include additional variables such as a patients baseline ambulatory status, American Society of Anesthesiologists (ASA) score, and their coronavirus disease 2019 (COVID-19) status on hospital admission [18-20].

The purpose of this study is to determine whether the inclusion of two additional modifiable clinical variables, BMI and smoking status, would further improve the predictive capacity and risk stratification regarding inpatient mortality for geriatric and middle-aged patients treated for hip fractures. We hypothesize that the addition of these modifiable risk factors will improve predictive capacity.

This is a retrospective cohort study. An Institutional Review Board-approved trauma database was queried for all patients aged 55 and older who sustained a low-energy hip fracture (low energy defined as a fall from standing or from a height of fewer than two stairs) between October 2014 and August 2021. All patients were treated at one urban academic medical center. Fracture patterns included in our analysis were subtrochanteric, femoral neck, or intertrochanteric hip fractures [AO Foundation/Orthopaedic Trauma Association fracture classifications: 31A, 31B, 32(A-C)].Patients were excluded if they were younger than 55 years old or had a high-energy mechanism of injury.

Each patients chart was reviewed for demographics that included age, BMI, gender, smoking status, baseline ambulatory status, and comorbidities using CCI. Smoking status was recorded as a current every-day smoker, former smoker, or never smoker. On a pre-study analysis, as current and former smokers were found to have no differences in complication, hospital quality, or mortality rates, these patients were grouped for analysis. Therefore, patients were considered smokers if they were current or former smokers. BMI was defined as underweight (<18.5 kg/m2), healthy weight (18.5-24.9 kg/m2), overweight (25.0-24.9 kg/m2), or obese (>30 kg/m2). Injury presentation variables collected were GCS scores and Abbreviated Injury Severity scores (AIS) for both the Head/Neck (AIS H/N) and Chest (AIS C).

Hospital quality measures collected were the length of stay (LOS) in days, the need for admission to the Intensive Care Unit (ICU), and discharge home (home was defined as either home independently or home with a health service). Mortality measures collected included inpatient and 30-day mortality. Inpatient complications recorded during each patients admission included sepsis/septic shock, pneumonia, deep vein thrombus/pulmonary embolism (DVT/PE), myocardial infarction (MI), acute renal failure/acute kidney injury (AKI), stroke, surgical site infection (SSI), decubitus ulcer, urinary tract infection (UTI), acute respiratory failure (ARF), anemia, and cardiac arrest.

Patients were initially grouped based on their smoking status, smokers (current and former) versus non-smokers (never smokers), and BMIs. Comparative analyses were conducted between each of these cohorts.For each patient, the baseline STTGMA score for hip fractures (STTGMAHIP_FX_SCORE) was calculated. The model was then adapted to include a patients BMI and smoking status (current every-day smoker, former smoker, or never smoker). A new mortality risk score, STTGMA_MODIFIABLE, was calculated for each patient. The predictive ability of each model was then compared using DeLongs test to assess the area under the receiver operating curves (AUROCs). Then, patients were stratified into risk quartiles based on their new respective STTGMA_MODIFIABLE mortality risk scores. Comparative analyses were conducted on each risk quartile to assess the efficacy of the new BMI and smoking status factors.

The following statistical tests were used as appropriate: Mann-Whitney U tests, chi-square tests, independent-sample t-tests, and analysis of variance (ANOVA). All statistics were calculated using SPSS Version 25 (IBM Corp., Armonk, NY, USA). The significance for this study was defined with an alpha of 0.05.

In total, 2,421 patients met the inclusion criteria. Characteristics for the total cohort were as follows: 69% of patients were female, the mean age was 80.7 10.2 years, mean BMI was 24.17 4.94 kg/m2, median GCS score was 15 (interquartile range (IQR): 0), mean CCI was 1.49 1.73, mean AIS Head/Neck was 0.03 0.27, and mean AIS Chest was 0.02 0.19.The majority of patients were White (71.71%). At baseline, most patients were community ambulators (67.91%), while 28.17% of patients were household ambulators, and 3.92% were non-ambulatory (Table 1).

An initial comparison of the current versus former smoker cohorts demonstrated that there were no differences in complication risk, hospital quality measures, or mortality outcomes (p > 0.05 for all). Subsequently, former and current smokers were grouped for further analysis. When comparing the smoker versus non-smoker cohorts, patients who were currently smoking or had a history of smoking experienced higher rates of inpatient (2.85% vs. 1.52%, p = 0.025) and 30-day (5.60% vs. 3.88%, p = 0.048) mortality. They also had higher rates of MI (2.01% vs. 0.76%, p < 0.01) and ARF (6.98% vs. 3.39%, p < 0.01), and had a longer inpatient LOS (in days: 6.82 4.83 vs. 6.28 4.17, p = 0.037) (Table 2).

Comparison among BMI cut-off cohorts demonstrated that underweight patients experienced higher rates of pneumonia (p = 0.033), decubitus ulcers (p=0.046), and need for the ICU (p < 0.01) (Table 3).

When comparing each risk scores respective AUROC, STTGMA_MODIFIABLE was found to improve the predictive capacity for inpatient mortality compared to STTGMAHIP_FX_SCORE (0.792 vs. 0.672, p = 0.0445) (Figure 1).

Regression weighting showed a coefficient of 0.337, with current smokers having the greatest absolute effect size (current every-day smoker = 1.011, former smoker = 0.674). While our other study findings demonstrate being below a healthy body weight increases the risk of inpatient mortality, BMI had a regression coefficient of 0.116, suggesting that a very high BMI is more strongly positively correlated with a higher risk of inpatient mortality. While this demonstrates statistically that a higherBMI positively correlates with a higher risk of inpatient mortality, our additional study findings demonstrate being below a healthy body weight similarly increases the risk of inpatient mortality.

When comparing risk quartiles for STTGMA_MODIFIABLE, multiple outcomes had significance. For mortality, patients in the highest risk quartile (STTGMA score >2.50%) experienced the highest rates of both inpatient (p < 0.01) and 30-day (p < 0.01) mortality. Patients in the highest risk cohort similarly experienced a longer inpatient LOS (p < 0.01), higher rates of sepsis (p < 0.01), pneumonia (p < 0.01), DVT/PE (p = 0.015), MI (p = 0.032), AKI (p < 0.01), ARF (p < 0.01), anemia (p < 0.01), cardiac arrest (p < 0.01), need for ICU level of care (p < 0.01), and were the least likely to be discharged home (p < 0.01) (Table 4).

The purpose of this study was to assess if the addition of various modifiable risk factors, a patients BMI and smoking status, to a validated inpatient mortality risk assessment tool improved the models predictive capacity and ability to effectively triage geriatric and middle-aged patients treated for hip fracture. This study demonstrates that the addition of these modifiable risk factors provided an improved predictive model. This improved mortality risk model will help guide treatment decisions and provide valuable prognostic information to discuss expectations surrounding patients injuries and potential outcomes with patients and their families.

This study demonstrated that patients who are either current smokers or have a history of smoking are at a higher risk for perioperative complications and potentially worse outcomes. While the higher mortality rate cannot be linked solely to a patients smoking status, the higher mortality rates found in our study align with those reported in the literature [11,12].The higher rates of MI and ARF seen in smokers can be expected as well due to the well-documented cardiovascular and pulmonary diseases found in patients secondary to smoking history [21,22]. Longer hospitalizations may also be attributed to the higher complication rates as patients in the smoker cohort required extended hospital stays to improve their health status before discharge. Similarly, these patients had worse baseline statuses prior to the injury, potentially necessitating a longer inpatient course. In addition, it is well documented in the literature that smoking delays wound healing [23-25]. For patients who required surgery as a part of their treatment for hip fracture, it is possible that they needed a longer time to heal due to the detrimental wound healing effects caused by smoking. While in our study, the rate of decubitus ulcer was higher in the smoker cohort, it was not significant. This may be due to the size of our patient cohort; given a larger patient cohort, we may have seen higher rates of decubitus ulcers. In addition, we did not capture the rate of wound infections which could also impact LOS. While the causes of the higher perioperative and mortality rates are multifactorial, smoking likely played a role. Smoking cessation has been proven to improve underlying cardiovascular and pulmonary health [26]. Providers may use this knowledge to counsel patients on the importance of both smoking cessation and/or continuing to remain smoke-free.

This study also demonstrated that patients who are underweightare at a higher risk for perioperative complications and potentially worse outcomes. Patients with a BMI of less than 18.5 kg/m2 were found to be at higher risk for pneumonia, decubitus ulcers, and the need for the ICU. Patients who are underweight may be malnourished and have vitamin deficiencies that impact immune function and wound healing, placing them at higher risk for skin breakdown. An international pressure ulcer prevalence survey and a study by Hyun et al. found that underweight and extremely obese patients were at higher risk for pressure ulcers [27]. Several studies have shown that the risk of infection, such as pneumonia, follows a U-shaped curve, suggesting that both underweight and obese patients are at higher risk [28,29]. While we saw a higher risk of pneumonia in the underweight cohort, it is possible that in our study, by not further splitting super-obese patients from obese patients, we did not see a higher risk of pneumonia in the higher BMI group. Additionally, while our study found that patients who are below a healthy body weight also had a higher risk of inpatient mortality, our regression showed that the higher a patients BMI, the higher the risk of inpatient mortality, similar to that found in the literature [8]. Obesity is associated with several comorbidities such as diabetes, heart disease, and increased risk for stroke, all health issues that have higher rates of mortality [27,30]. Patients who are underweight or obese can be identified as higher risk on arrival, allowing for timely intervention and appropriate medical management. Prior to discharge, counseling can be provided on effective nutrition plans and active lifestyle adjustments to help patients attain healthy body weight.

The inclusion of these modifiable risk factors in the STTGMA tool allowed for effective triage of high-risk patients into appropriate risk quartiles. Stratification using STTGMA_MODIFIABLE identified patients who experienced not only higher rates of inpatient mortality, 30-day mortality, and the need for the ICU but also higher rates of serious inpatient complications such as sepsis, DVT/PE, MI, and AKI. Providers may use these added clinical variables to better identify patients who may require more intensive medical management and timely intervention. This may have implications to improve outcomes and reduce hospital costs by proactively managing patients to help lower complication and mortality rates while shortening hospital admissions.

This study has several limitations. First, as a retrospective study, it is subject to the common biases associated with this study format. Second, our analysis relied upon database entries for a patients smoking status. Therefore, we were unable to assess a patients smoking status if it was not recorded in the electronic medical record (EMR). However, as only 30 patients did not have a smoking status EMR entry, the impact of this limitation may be minimal. Third, our study did not include the number of cigarettes, packs, and pack-years for each patient. As the adverse effects of tobacco smoking may have a dose-dependent relationship, our analysis could not account for this component of a patients smoking status. Further analysis may be done to include a weighting factor that considers smoking amount. Fourth, our study did not distinguish super-obese patients from obese patients. There may be an additional risk or protective factors associated with super-obese patients. Additional studies may also be conducted to include a cost analysis to assess the impact of different BMIs and smoking status on hospital costs. Lastly, future studies may focus on a prospective analysis comparing mortality risks overtime in a cohort that modifies its risk (i.e., by losing weight or stopping smoking) versus a cohort that does not.

Smoking, obesity, and being below a healthy body weight increase the risk of perioperative complications and poor outcomes. Including smoking and BMI improves the STTGMAHIP_FX_SCOREtool to predict mortality and risk stratify patient outcomes. Because smoking, obesity, and being below a healthy body weight are modifiable patient factors, providers can counsel patients and implement lifestyle changes to potentially decrease their risk of longer-term poor outcomes, especially in the setting of another fracture. For patients who are former smokers, providers can use this information to encourage continued restraint and healthy choices.

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The Role of Smoking and Body Mass Index in Mortality Risk Assessment for Geriatric Hip Fracture Patients - Cureus

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Adverum cuts jobs, restructures to give eye gene therapy another shot – BioPharma Dive

Posted: July 11, 2022 at 2:38 am

Dive Brief:

Despite significant setbacks that have left the fate of its eye gene therapy in doubt and shares trading near all-time lows, Adverum hasnt given up.

The company is one of a few gene therapy makers aiming to develop a one-time treatment for diabetic macular edema and age-related macular degeneration, two common forms of vision loss that are treated with chronic injections of biologic medicines. But those drugs, like Eylea and Lucentis, are highly effective and considered safe, making the bar much higher for a gene therapy whose main goal is to improve convenience.

Adverums program was also beset by side effects the company once described as not seen before in ocular gene therapy, a combination of inflammation, vision loss and decrease in eye pressure observed in five trial participants.

Adverum stopped that trial, in diabetic macular edema, in 2021. At the time, some analysts suggested the company should attempt a reverse merger, a way for struggling biotechs to bring in new assets by combining with a privately held company seeking fast access to the public markets.

The company instead vowed to press on. Executives suggested testing a lower dose than previously planned with a different regimen of protective drugs could lead to better results in AMD. In 2021, the company noted that no cases of severe inflammation were observed in DME patients treated with a lower dose or in participants with AMD in another trial.

Adverum has since gained clearance from U.S. regulators for its new plan, a Phase 2 trial in AMD thatll test both the lowest dose evaluated in previous studies as well as one more than three-times lower. With shares trading at just over $1 apiece and equity harder to raise during the sectors downturn, Adverum has turned to cost-cutting to save money and fund the work. The savings could enable the company to get to one-year results from that trial, in 2023, without needing to raise more cash, wrote RBC analyst Luca Issi.

Yet Adverums odds remain long. A rival gene therapy from Regenxbio is already in Phase 3 testing in AMD, and pending positive results, could lead to an approval filing in 2024. The company remains a show-me story given its history, Issi wrote. Additionally, Adverums decision to turn to layoffs, rather than a partnership, may also signal limited strategic interest in the platform, he added.

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One-time gene therapy injection could provide HIV treatment that may last a lifetime – OHSU News

Posted: July 11, 2022 at 2:38 am

Jonah Sacha, Ph.D., at OHSU's Oregon National Primate Research Center and Vaccine & Gene Therapy Institute. (OHSU/Kristyna Wentz-Graff)

A new pre-clinical study in nonhuman primates will evaluate an experimental drugs potential use as a gene therapy that could prevent people who have HIV from having to take daily antiviral drugs for the rest of their lives.

The research will be led by Oregon Health & Science University researcher Jonah Sacha, Ph.D., who also serves as a scientific adviser to CytoDyn, the biotechnology company developing the drug, called leronlimab. The study is funded by a five-year grantof up to $5 million that was recently awarded to OHSU by the National Institute of Allergy and Infectious Disease, which is part of the National Institutes of Health.

This grant will fund the development and early study of leronlimab as a potential single-injection gene therapy, said Sacha, professor at OHSUs Vaccine and Gene Therapy Institute and Oregon National Primate Research Center. If this approach works as hoped, it could provide a functional cure for HIV, meaning it could suppress HIV enough that patients would no longer need to take daily antiviral pills for the rest of their lives."

In an earlier study, Sacha and colleagues found leronlimab completely preventednonhuman primates from being infected with the monkey form of HIV. That result indicated leronlimab held promise as a potential pre-exposure drug to prevent human infection from the virus that causes AIDS.

Now, this study aims to design a way to offer leronlimab as gene therapy. Sacha and colleagues will explore how to contain the coding sequence of the experimental drug inside a lab-made form of the adeno-associated virus, an approach that gene therapy researchers call an AAV vector. The resulting therapy would then be injected inside the body where muscle cells would make leronlimab long term.

Leronlimab is a monoclonal antibody that blocks HIV from entering immune cells through a surface protein called CCR5. The drug has demonstrated it can mimic a CCR5-deficient donor by occupying all available CCR5 molecules, but this would require a new method for delivery as a gene therapy. Viral vectors have been used to deliver antigens from specific pathogens for decades.

In this project, researchers will design a synthetic AAV vector to enable the long-term production leronlimab inside the body. The goal is to develop a safe and effective single injection that suppresses HIV replication and eliminates the need for life-long antiretroviral therapy.

Currently, patients often take multiple antiretroviral pills daily, said Sacha. Removing this pill burden could not only improve patients quality of life, but remove problems with adherence.

Rhesus macaques at OHSUs Oregon National Primate Research Center that have been exposed to a monkey version of HIV will be given a single AAV injection that contains leronlimab. Researchers will monitor the nonhuman primates for years to assess the safety and efficacy of this approach.

This research is supported by the National Institute of Allergy And Infectious Diseases of the National Institutes of Health under Award Number R01AI166969. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

In our interest of ensuring the integrity of our research and as part of our commitment to public transparency, OHSU actively regulates, tracks and manages relationships that our researchers may hold with entities outside of OHSU. In regard to this research, Dr. Sacha has a significant financial interest in CytoDyn, a company that may have a commercial interest in the results of this research and technology. Reviewdetails of OHSU's conflict of interest programto find out more about how we manage these business relationships.

All research involving animal subjects at OHSU must be reviewed and approved by the universitys Institutional Animal Care and Use Committee (IACUC). The IACUCs priority is to ensure the health and safety of animal research subjects. The IACUC also reviews procedures to ensure the health and safety of the people who work with the animals. No live animal work may be conducted at OHSU without IACUC approval.

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Can Philly’s Developers Shepherd Cell And Gene Therapy Startups Through The ‘Valley Of Death’? – Bisnow

Posted: July 11, 2022 at 2:38 am

The life sciences real estate market in Philadelphia is coming into its own just as its cell and gene therapy industries stare down their first economic cycle with high inflation and spiking federal interest rates.

Bisnow/Virginia Baker

Mosaic Development Partners founder and co-owner Greg Reaves and Brandywine Realty Trust Senior Managing Director Jeff DeVuono at Bisnow's Philly Life Sciences Summit event at Budd Bioworks on June 28, 2022

Despite the success of Spark Therapeutics, the vast majority of cell and gene therapy candidates remain in early stages of testing, with relatively few in clinical trials and only a handful commercially available. So much of the public and private sector in the area is focused on sustaining the growth of the past few years, but both are being called on to shepherd startups through the valley of death, or the period between early stage trials and commercialization when funding is hardest to come by and most desperately needed.

One of the costs that balloon during that period is real estate, as the space a biotech startup requires for advancing a therapy through testing phases is often what takes it from a couple of stations at an incubator to its first proper lease. But that graduation space, as the industry calls it, is in extremely short supply across the Philadelphia region, Longfellow Real Estate Partners Managing Director of Research Lauren Gilchrist said at Bisnows Philadelphia Life Sciences Summit on June 28.

Our incubator spaces and our [life sciences] coworking spaces are filling up with companies that are probably ready for their graduation spaces, and in other markets would be in graduation space, Gilchrist said. But we have not yet provided that space and the right continuum of services in Philadelphia.

While several ground-up construction projects are underway, the two most significant adaptive reuse projects in and around Center City 401 North Broad St. and The Curtis are filling up space virtually as soon as they can convert it.

To meet the timeline specific to life sciences tenants, Philadelphia developers have had to get comfortable with starting buildings on speculation, with multiple such projects underway inUniversity City and the Philadelphia Navy Yard. One uCity Square, set to deliver this fall, is up to 80% leased, said Wexford Science + Technology Director of Development Pete Cramer, whose company is developing the building in partnership with the University City Science Center and Ventas. But the upfront cost and risk associated with speculative development keep rising.

Bisnow/Virginia Baker

Longfellow Real Estate Partners Managing Director of Research Lauren Gilchrist and Delaware Innovation Space founder and CEO Bill Provine

With financing costs rising precipitously amid higherinterest rates, capital markets activity is cooling in commercial real estate, all while inflation and supply chain snags persist to make projects more expensive and more unpredictable in terms of cost. But if a company has the money, spending it now seems to be the order of the day, panelists at the event agreed. Even if specialized equipment is more expensive than it has been for years, future price hikes and surprise inventory disruptions are being treated as a virtual certainty across the industry.

The joint venture of Ensemble Real Estate Investments and Mosaic Development Partners tasked with leading the Navy Yards next wave of development, which has one spec lab building under construction, plans on front-loading more of its capital expenditures for its next building, which is planned for Current Good Manufacturing Practices-compliant biomanufacturing, Mosaic founder and co-owner Greg Reaves said at the event.

The GMP that you knew before Covid is not the same GMP [as today], Reaves said. Purchasing materials upfront is a requirement today, and I dont think you can avoid that. If you expect to be open anytime in 2023 or 2024, you have to be thinking seriously about moving forward on risk.

To build on speculation is to rely on the Field of Dreams line oft-quoted in real estate, If you build it, they will come. But in a sector where the majority of tenants are pre-revenue, selecting a tenant from a pool of interested parties is an exercise in risk assessment. Multiple landlords and brokers at the full-day event stressed the importance of believing in a tenants research or, more importantly, its leadership.

A lot of these early stage companies can't scale because they don't have the expertise, they don't have the network and they don't know what they don't know, HDR Managing Principal Elizabeth Mahon said. In the cell and gene therapy world, there's not enough leadership talent to go around. So when you're dealing with a lot of technical founders, scientific founders, first-time CEOs and [chief science officers], you're really limited because they're learning on the fly.

Bisnow/Virginia Baker

University Place Associates President Anthony Maher, Center for Breakthrough Medicines co-founder Audrey Greenberg and Tactix Real Estate Advisors Senior Broker Gary Zoloff

Having a leader with deep experience or a wide network is crucial for a life sciences startups appeal to landlords, not least because it gives those startups an edge in recruiting venture capital investors. In situations when a landlord may believe in a tenant even if the latter lacks its own access to capital, some landlords are tapping their own capabilities or partnerships.

University Place Associates has connected potential tenants with its capital partners, the joint venture of Cantor Fitzgerald and Silverstein Properties that has financed 3.0 University Place, set to deliver next year, and with which UPA is negotiating financing terms for the following 4.0 University Place next door, UPA President Anthony Maher said.

My guess is that [a startups] investors are not looking at this as a gulp moment of hyper-leverage, Maher said. They're looking at it as a serious way to get these companies into occupancy and production by paying rent and other fees and moving along in the life cycles of their businesses.

The Center for Breakthrough Medicines, which operates both an accelerator and a contract development and manufacturing organization at the Discovery Labs complex in King of Prussia, uses a milestone-based lease structure with some promising companies, CBM co-founder Audrey Greenberg said. Under that system, CBM initially leases space at cost to startups, andleases grow in cost commensurate with the funding and/or revenue those startups wind up bringing in.

CBM announced in late June that it plans to expand its facility by 1M SF, including 90K SF of cGMP space, with a focus on cell therapy. That expansion, as well as CBMs leasing model, is made possible by a $350M equity investment the facility received at the start of the year from SK Inc., the investment arm of South Korean conglomerate SK Group. That investment was announced only a few weeks before the Russian invasion of Ukraine, which the Federal Reserve cited as an inflationary factor in raising its interest rate, which in turn has made capital more expensive.

We have to acknowledge that pricing has changed, Greenberg said. I think that more can be done in terms of taking risk on the tenant base and underwriting credit in a creative way. But it's a challenging time to do that. So you know if it was last year or the year before, it might be different.

Bisnow/Virginia Baker

Hargrove's Justin Pagliaro, Breakthrough Properties' Joe Traynor, Colliers' Joseph Fetterman and Plymouth Group's Michael Davis at Bisnow's Philly Life Sciences Summit event, hosted at Plymouth's Budd Bioworks development in June 2022.

Panelists throughout the day pleaded for more assistance from state government. The commonwealth of Pennsylvanias Redevelopment Assistance Capital Program served as a small-yet-key financing piece for tenant space at 3.0 University Place and 401 North Broad, Maher said. A RACP-type program specific to life sciences, or private capital deployed in a similar way, would go a long way toward stabilizing many companies slogging through the biotech valley of death.

A backstop for a non-credit tenant that you believe in, or [funding] an incubator, could go a long way, Maher said. I can show you [potential tenants] that could stabilize on revenue. But you have a blip in the first six months, then you have a blip again at month 18, someone needs to fill that void. And the ask would not be $50M, it would be $5M.

Massachusetts and California created programs that filled crucial financing gaps in the past couple of decades, and their life sciences hubs are better for it, Mahon said. But part of the difficulty facing the Philadelphia market is that its suburbs are split across multiple states, introducing a competitive factor that ultimately works against the region on the whole, Delaware Innovation Space founder and CEO Bill Provine said. But New Jersey Gov. Phil Murphy has not let the presence of New York in the north and Philly in the south get in the way of offering economic incentives to companies through the New Jersey Economic Development Authority.

It's not just the state, every level has to be engaged in this, Philadelphia Department of Commerce Senior Director of Business Development Sam Woods-Thomas said. And that's why you see places like Massachusetts, New York and California eating our lunches, frankly. We hope that won't continue. We're going to work hard to make sure that doesn't continue.

Though it only represents a part of the regions ecosystem and has a much smaller budget, the city of Philadelphia is working on a way to address the problem of institutional support, Woods-Thomas said.

In the next year or so you're going to see policies and incentives coming from us that begin to make a lot more sense than what we have right now, he said.

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AAVIATE: Gene therapy via suprachoroidal drug delivery may lower treatment burden for patients with AMD – Modern Retina

Posted: July 11, 2022 at 2:38 am

Emily Kaiser [EK]: Hello, welcome. I'm Emily Kaiser, editor of Modern Retina. I'm sitting down with Dr. Rahul Khurana to discuss the Aaviate study. Dr. Khurana, can you tell us a little bit about the Aaviate data presented at the Angiogenesis meeting?

Rahul Khurana, MD [RK]: For sure, Emily.

Aaviate is a really exciting clinical trial that I've been involved with and at Angiogenesis this year, we presented the updated data on the first two cohorts. And so to give everyone kind of a background, obviously, gene therapy is a very exciting area of interest right now.

We know there's a lot of unmet needs in our treatments of macular degeneration. You know, these treatments often are very effective, but they require a lot of treatments. And there's a high burden with our current set of treatments out there, and gene therapy offers the potential for a one-time treatment to give us long-term, anti-VEGF suppression and really a long-term answer to this kind of chronic disease.

And so Aaviate is along with many gene therapy programs, or studies, that are looking to kind of tackle this. And the thing which is interesting about Aaviate is that most of gene therapy has really looked at it the traditional approaches. Either theyve gone intravitreal, which is something we're very used to because we do these injections, or done a subretinal gene therapy delivery, which requires surgery, which is much more invasive. And Aaviate utilizes a suprachoroidal approach.

And the benefit of this is that we get good drug delivery of inhibiting anti-VEGF gene therapy, but by delivering it in the office. And one of the benefits of suprachoroidal delivery over intravitreal delivery is that in intravitreal, there is a potential for a lot of exposure of the medicine to not just the back of the eye, but also the front of the eye. And we've seen in some early gene therapy programs, a lot of complications involving inflammation and hypotony.

In a suprachoroidal approach, you can get a very high concentration to the retina with very low concentration to the anterior segment.

EK: That's really interesting. Has anything developed since the presentation?

RK: the study has been ongoing. So what I presented at Angiogenesis was the first two cohorts in the sense that those patients had been fully enrolled, and we had up to date up to six months. So that was really exciting. And we'll kind of delve into some of the details there. But there's still cohorts three, four, and five, which are now fully enrolled. So since that time, we've now fully enrolled those patients. And we're basically waiting to hear back on updates, or we were waiting to hear back those results. And we need to once they're fully enrolled, we need to have the subsequent time to see how these patients do.

EK: And what are the next steps?

RK: Part of the next steps for gene therapy is really to finish up the clinical study. The patients are all enrolled, which is wonderful. Now we want to see how they did in these higher enrolling cohorts. So one thing that we haven't talked about is what were the results that we actually found from the first two cohorts? And so as I mentioned before, Aaviate takes patients who have been previously treated so these patients who were in the study were basically patients who needed to get multiple injections.

On average, they average nearly nine injections in the previous year, which is about an injection every five weeks. And we took those patients who basically needed regular anti-VEGF therapy, and we basically offered them a super coronal injection of RGX-314, which involves a novel Aaviate vector, which encodes for an anti VEGF monoclonal antibody fragment, which is transduced, or basically transvexed, the patient's own retinal cells to produce anti-VEGF protein to effectively give you long term suppression.

And the data showed that in the first two cohorts where this was done, not only was the treatment quite safe, there was a very low rate of inflammation and no serious adverse events. But more impressively, that the number of treatments had gone down dramatically. The patients in the study were able to maintain their visual acuity, which was wonderful to see. But more importantly, the number of injections went down significantly.

As I told you, before, most of these patients needed about an injection every five weeks, and in the study, the number of injections went down nearly 70 to 79% than they had before receiving the gene therapy, they were able to maintain the visual acuity, maintain the retinal anatomy, and the number of patients who didn't even need injections was nearly 30% in the first cohort, and nearly 40% in the second cohort. And that was quite exciting because this truly was kind of delivering on the promise of a once-and-done therapy. But as I said before, we really need the long-term data to kind of see how this translates and also we need to see how higher doses if we can get better efficacy and also maintain a very good safety profile.

EK: Wow. So what does this mean for clinicians and for patients?

RK: I think it offers a really exciting hope for both our patients and physicians. As we mentioned before, we have a lot of treatment options for anti-VEGF therapy and they do work very well. The problem is that they require a lot of treatments and there's a high treatment burden, and this is challenging for patients because not all patients can come back in there's a high rate of lost to followup, non-compliance, and non-adherence to the treatment regimens. And we've seen in Phase 3 clinical studies, especially in follow up in real world practice that when patients are not getting regular treatments, they lose vision. And that's why we've it's been hard to replicate the excellent results we've seen in the Phase 3 studies in real world practice. And the hope is that if one of these gene therapy treatments can work, we can offer a really one-and-done or a much more sustainable treatment therapy for our patients, which ultimately lead to better compliance and better visual outcomes.

EK: Fantastic. Well, thank you so much for the update.

RK: My pleasure. Thanks for having me.

Note: This transcript has been lightly edited for clarity.

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Myrtelle Announces Positive Data for Its investigational Proprietary rAAV-Olig001-ASPA Gene Therapy in Canavan Disease at the National Tay Sachs &…

Posted: July 11, 2022 at 2:38 am

WAKEFIELD, Mass.--(BUSINESS WIRE)--Myrtelle Inc. (Myrtelle or the Company), a gene therapy company focused on developing transformative treatments for neurodegenerative diseases, today announced in a presentation by Armen Asatryan, MD, Chief Medical Officer, at the National Tay Sachs and Allied Diseases Annual Family Conference held in Denver, Colorado, that the Companys recombinant adeno-associated virus (rAAV) vector-based investigational gene therapy for Canavan disease (CD) has shown encouraging early efficacy data and a favorable safety profile in the first three patients treated through their six month visit post-treatment in the Companys open-label Phase 1/2 First-in-Human (FIH) clinical trial at Dayton Childrens Hospital (Dayton, Ohio). Myrtelles FIH trial utilizes the Companys proprietary rAAV vector to directly target oligodendrocytes, the affected brain cells in CD responsible for producing myelin the insulating material that enables proper neuronal function. In CD, the production of myelin is affected due to a mutation in the Aspartoacylase gene (ASPA) encoding the enzyme Aspartoacylase (ASPA). The oligodendrocyte-targeting rAAV vector-based gene therapy is intended to restore ASPA function, thus enabling metabolism of N-Acetylaspartate (NAA), a neurochemical that is abundant in the brain, and thereby supporting myelination. Five patients have been treated in the ongoing Phase 1/2 study with favorable safety and tolerability results to date and no study drug-related adverse events.

Analyses of the first three patients at 6-months post-treatment by Magnetic Resonance Imaging (MRI) demonstrated increases in white matter and myelin in the brain. Improvements were also exhibited in scores on the Gross Motor Function Measure (GMFM) and Mullen Scales of Early Learning (MSEL), validated functional scales. Observed improvements contrast the continuous clinical deterioration expected with the natural progression of the disease.

The 6-month functional and anatomic data observed in the first three patients elicit hope when we compare it with the natural course of this devastating disease. Equally encouraging are positive changes in the patients reflexes, eye tracking, vocalizations to communicate, and overall awareness said Robert Lober, MD, PhD, FAANS, Co-Principal Investigator and Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine and Attending Neurosurgeon at Dayton Childrens Hospital in Dayton, OH. These improvements suggest the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease.

Per the FIH trial protocol, the gene therapy is administered as a single total dose of 3.7 x 1013 vg delivered by intracerebroventricular (ICV) injection to target the oligodendrocytes of the brain where ASPA activity is deficient in CD patients.

Myrtelles current investigational gene therapy targeting oligodendrocytes with a unique rAAV vector builds upon over two decades of science and effort with the potential, if successful, to usher in new treatment options for children with Canavan disease, commented Armen Asatryan, MD, MPH, Chief Medical Officer of Myrtelle. We intend to build on these encouraging initial findings and complete our current Phase 1/2 clinical study, following which we plan to engage with regulatory authorities to advance the program to the later stages of clinical development.

ABOUT MYRTELLE

Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Companys website at: http://www.myrtellegtx.com.

ABOUT CANAVAN DISEASE

Canavan disease (CD) is a fatal childhood genetic brain disease in which mutations in the Aspartoacylase gene (ASPA) prevent the normal expression of Aspartoacylase (ASPA), a critical enzyme produced in oligodendrocytes that breaks down the neurochemical N-Acetylaspartate (NAA). When not properly metabolized by oligodendrocytes, NAA accumulates in the brain and negatively affects bioenergetics, myelin production, and brain health. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available.

More information on Myrtelles clinical study in Canavan disease can be found on https://clinicaltrials.gov/ under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com.

Forward-Looking Statements

This press release contains forward-looking statements. Words such as may, believe, will, expect, plan, anticipate, estimate, intend and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding encouraging early data support further development of rAAV-Olig001-ASPA as a potential therapeutic approach for children diagnosed with Canavan disease, the improvements suggesting that the gene therapy and the route of administration are directly targeting the oligodendrocytes, the key cells affected in Canavan disease, the potential of Myrtelles current investigational gene therapy targeting oligodendrocytes, to usher in new treatment options for children with Canavan disease. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based in information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelles program demonstrating safety and efficacy, as well as results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelles expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events or otherwise.

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