An international study led by Paolo Fiorina from Boston Children's Hospital and in collaboration with researchers from University of Milan highlights the beneficial effect of inhibiting the death receptor TMEM219 to restore mucosal healing in inflammatory bowel diseases. Findings were published today in the Journal of Clinical Investigation. Fiorina and colleagues demonstrated that the pharmacological blockade of TMEM219 signal through a recombinant protein based on the extracellular portion of the TMEM219 receptor is able to preserve self-renewal ability of intestinal stem cells in inflammatory bowel disease, protect intestinal stem cells from cell death induced by TMEM219 activation, and prevent colitis development in mouse models. These results were confirmed by the selective genetic inhibition of TMEM219 on intestinal stem cells LGR5 in vivo which allowed for the preservation of mucosal regeneration and healing in inflammatory bowel disease. This may thus represent a new mechanism of disease in which TMEM219 overactivation induces intestinal stem cell death and prevents mucosal renewal during inflammation. This new signaling is able to control the fate of intestinal stem cells and modulate their survival. The study shows how this new mechanism of disease, which is overactive in the intestine during inflammation, is able to control mucosal self-renewal, particularly in inflammatory bowel disease. The dysregulation of this axis in patients with IBD and non-responding to therapy or with active disease suggests that TMEM219 signaling may prevent mucosal healing and therefore exerts a detrimental effect that aggravates the disease condition." This study goes further and identifies the TMEM219 signals as a novel mechanism of disease in colitis. "The possibility of re-establishing self-renewal abilities of intestinal stem cells and promote mucosal healing during colitis is of extraordinary importance for patients with inflammatory bowel disease, especially in those not responding to current therapy in whom the disease is relapsing frequently, and surgery is at the end required," says Fiorina. TMEM219 signaling promotes intestinal stem cell death and exacerbates colitis. In this interview, industry expert Dr. Lohit Khera discusses the evolving role of microRNA in research, diagnostics, and precision medicine, highlighting the latest innovations in RNA extraction and analysis Learn how experts are advancing benzodiazepine analysis and detection using insights from the lab. News-Medical.Net provides this medical information service in accordance with these terms and conditions. Please note that medical information found on this website is designed to support, not to replace the relationship between patient and physician/doctor and the medical advice they may provide. Hi, I'm Azthena, you can trust me to find commercial scientific answers from News-Medical.net. Registered members can chat with Azthena, request quotations, download pdf's, brochures and subscribe to our related newsletter content. A few things you need to know before we start. While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles. Please do not ask questions that use sensitive or confidential information.

The US Food and Drug Administration (FDA) has granted accelerated approval to telisotuzumab vedotin-tllv (Emrelis, AbbVie) for locally advanced or metastatic nonsquamous non–small cell lung cancer (NSCLC) with high c-Met protein overexpression after prior systemic therapy. The FDA also approved the VENTANA MET (SP44) RxDx Assay (Roche Diagnostics) as a companion diagnostic to detect high c-Met protein overexpression, defined as 50% or more of tumor cells with 3+ staining. Telisotuzumab vedotin-tllv “is the first and only treatment approved for previously treated advanced NSCLC patients with high c-Met protein overexpression who often face poor prognosis and have limited treatment options,” AbbVie said in a press release. C-Met is a cell surface protein that is overexpressed in approximately 25% of patients with advanced epidermal growth factor receptor (EGFR) wild-type, nonsquamous NSCLC, driving tumor progression. Approval was based on the phase 2 LUMINOSITY trial in 84 patients with EGFR wild-type, nonsquamous NSCLC and high c-Met protein overexpression. Patients received telisotuzumab vedotin-tllv as monotherapy in the second or third-line setting. The most common adverse reactions, in 20% or more of study participants, were peripheral neuropathy, fatigue, decreased appetite, and peripheral edema. The recommended telisotuzumab vedotin-tllv dose is 1.9 mg/kg intravenously up to a maximum of 190 mg for patients who weigh 100 kg or more every 2 weeks until disease progression or unacceptable toxicity. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow.

A recent study published in Life Metabolism has uncovered a surprising new player in the body's ability to regulate blood sugar after eating. Researchers from Baylor College of Medicine and the University of Namur found that an enzyme called hyaluronidase-1 (HYAL1) plays a crucial role in suppressing excessive glucose production in the liver, particularly after meals. This discovery could open new doors for treating metabolic disorders like type 2 diabetes, where blood sugar control is impaired. The study focused on hyaluronan (HA), a sugar molecule that naturally surges in the bloodstream after eating. While HA is known for its roles in tissue structure and inflammation, this research reveals its unexpected connection to glucose metabolism. Normally, gluconeogenesis is suppressed after meals to prevent blood sugar spikes, but in diabetes, it remains overactive. Using genetically modified mice, the researchers found that deleting the Hyal1 gene led to higher glucose production, especially in mice fed a high-fat diet. Digging deeper, the team uncovered the mechanism: HYAL1's breakdown of HA redirects cellular Uridine diphosphate N-acetylglucosamine (UDP-GlcNAc), reducing a key modification (O-GlcNAcylation) on mitochondrial proteins that are key in energy metabolism. This lowers ATP production, making it harder for the liver to sustain glucose synthesis. Importantly, this regulation remains functional even in insulin-resistant conditions, making it an attractive target for therapies. With diabetes affecting millions worldwide, this research highlights an overlooked yet vital piece of the metabolic puzzle that could lead to new strategies for managing diabetes, such as enhancing HYAL1 activity or manipulating HA levels after meals. In this interview, industry expert Dr. Lohit Khera discusses the evolving role of microRNA in research, diagnostics, and precision medicine, highlighting the latest innovations in RNA extraction and analysis Learn how experts are advancing benzodiazepine analysis and detection using insights from the lab. News-Medical.Net provides this medical information service in accordance with these terms and conditions. Please note that medical information found on this website is designed to support, not to replace the relationship between patient and physician/doctor and the medical advice they may provide. Hi, I'm Azthena, you can trust me to find commercial scientific answers from News-Medical.net. To start a conversation, please log into your AZoProfile account first, or create a new account. Registered members can chat with Azthena, request quotations, download pdf's, brochures and subscribe to our related newsletter content. A few things you need to know before we start. Please check the box above to proceed. While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles. Please do not ask questions that use sensitive or confidential information.

A new study uncovers how working at 35,000 feet may disrupt DNA repair and immune function in flight attendants, offering fresh insights into the health risks faced by cabin crews and the urgent need for stronger protections. Study: Suppressed DNA repair capacity in flight attendants after air travel. An increased cancer risk and compromised reproductive health among flight attendants (FAs) could be due to impaired genomic stability and altered immune responses linked to their air travel. These workers are also exposed to many biological, physical, and psychosocial stressors through difficult interactions with passengers or quick turnaround times between flights. As compared to the general population, epidemiological studies have reported that FAs are at a greater risk of developing breast and skin cancer, as well as non-Hodgkin lymphoma, and experiencing adverse reproductive outcomes. However, the underlying mechanisms associated with this increased cancer risk and other health issues in FAs remain unclear. Previous studies have reported that environmental and occupational exposure to DNA-damaging agents can lead to transcriptional stress, cell death, genomic instability, stress, and senescence. Occupational hazards may also increase the risk of DNA damage by inducing inflammatory responses that produce endogenous reactive oxygen species (ROS). In humans, many DNA repair mechanisms, such as mismatch repair (MMR), base excision repair (BER), non-homologous end joining (NHEJ), homologous recombination (HR), and nucleotide excision repair (NER), are inherently present to protect the native genome from DNA-damaging agents. However, a defective DNA repair system increases the risk of several diseases, particularly cancer. This preliminary investigation hypothesized that altered DNA repair and immune responses are the potential underlying mechanisms responsible for increased cancer risk in FAs. To test this hypothesis, the effect of air travel on DNA damage and repair responses in FAs was assessed. DNA repair capacity (DRC) for DNA repair pathways, including NHEJ, BER, NER, MMR, and HR, was assessed using functional high-throughput, fluorescence multiplex-based host cell reactivation (FM-HCR) assays. FA blood samples were analyzed using an unprecedented method of DNA repair based on host cell reactivation technology in resting lymphocytes. Blood samples for BF measurements were collected five to 12 hours before flight departure from Boston Logan International Airport, and blood samples for AF assessments were collected within 1.5 hours of landing at the same airport. The duration between departure and return varied between 27 hours and 10 days, depending on each individual's flight schedule. This pilot study included a small sample of nine FAs, three of whom were male and six females between 24 and 64 years of age, were included in the research. The employment duration as an FA ranged from two to 41 years. There was no significant difference in the blood cell count landscape between the BF and AF samples. However, lymphocyte counts were significantly higher in AF samples, suggesting an inflammatory response associated with flight travel-related exposures. Higher basophil counts were also observed in AF blood samples; while a statistically significant increasing trend was noted in a subgroup of FAs (after excluding two individuals with notably high counts), the trend did not reach statistical significance when all nine participants were included due to high variability. Likewise, no statistically significant difference in the average basal DNA damage was observed between BF and AF samples. A statistically insignificant trend towards a marginal increase in the time needed to repair initial genomic DNA damage was observed in AF samples compared to BF samples using the CometChip assay. Thus, increased DNA damage could be due to impaired DNA repair, particularly linked to impaired single-strand breaks (SSBs) repair by BER processing, which may arise from oxidative DNA damage. Although most ionizing radiation (IR)-induced DNA damage was repaired within one hour, considerable heterogeneity in repair kinetics data was observed. The paper detailed that while some individuals' after-flight samples showed slower repair kinetics compared to their before-flight samples, others showed more rapid kinetics, highlighting inter-individual differences and varied responses to air travel. As compared to the BF samples, a significant reduction in the repair of oxidative DNA damage and IR-induced plasmid damage was observed in the AF samples. The findings from this pilot investigation, which involved a small number of participants, indicated alterations in blood cell count, decreased activity in repairing oxidative and IR-induced genomic lesions, and heterogeneity in repair kinetics in AF samples of FAs as compared to their BF samples. These observations suggest a potential mechanistic link where flight-related exposures might simultaneously promote inflammatory processes that increase oxidative DNA damage while also suppressing the DNA repair mechanisms that protect against such damage, contributing to genomic instability. These results should be interpreted with caution, given the study's scale. This initial study serves as the foundation for future large-scale studies on in-flight crew, which are needed to confirm these preliminary findings and may support the development of informative policies to protect FAs from unnecessary health risks. Priyom holds a Ph.D. in Plant Biology and Biotechnology from the University of Madras, India. Priyom has also co-authored several original research articles that have been published in reputed peer-reviewed journals. Please use one of the following formats to cite this article in your essay, paper or report: Air travel disrupts DNA repair in flight crew, raising cancer concerns. "Air travel disrupts DNA repair in flight crew, raising cancer concerns". "Air travel disrupts DNA repair in flight crew, raising cancer concerns". Air travel disrupts DNA repair in flight crew, raising cancer concerns. In this interview, industry expert Dr. Lohit Khera discusses the evolving role of microRNA in research, diagnostics, and precision medicine, highlighting the latest innovations in RNA extraction and analysis Learn how experts are advancing benzodiazepine analysis and detection using insights from the lab. News-Medical.Net provides this medical information service in accordance with these terms and conditions. Please note that medical information found on this website is designed to support, not to replace the relationship between patient and physician/doctor and the medical advice they may provide. Hi, I'm Azthena, you can trust me to find commercial scientific answers from News-Medical.net. Registered members can chat with Azthena, request quotations, download pdf's, brochures and subscribe to our related newsletter content. A few things you need to know before we start. Please check the box above to proceed. While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles. Please do not ask questions that use sensitive or confidential information.

A new clinical study reveals that daily black tea kombucha can promote healthier gut bacteria, especially in people with obesity, by reducing harmful microbes and supporting beneficial ones. Study: Regular Consumption of Black Tea Kombucha Modulates the Gut Microbiota in Individuals with and without Obesity In a recent study in The Journal of Nutrition, researchers investigated the intestinal health impacts of regular black tea kombucha. The study was carried out over eight weeks and comprised initially 46 participants, with 23 participants assigned to each of two categories: normal weight or obese. Study outcomes were measured by comparing participants' stool, urine, and blood samples between baseline (0 weeks) and completion (8 weeks of intervention). Study findings revealed that the kombucha contains 145 phenolic compounds (flavonoids = 81%, phenolic acids = 19%) and contributes to the growth of beneficial gut bacteria across both cohorts. Changes in gut fungal composition were also observed after the kombucha intervention, with an increase in the abundance of certain fungi like Saccharomyces, though overall fungal alpha-diversity did not generally increase across all groups and was noted to be higher at baseline in the obese group before potentially decreasing or changing by the end of the intervention. Obesity is a growing global health concern, afflicting more than 1 billion people today, and estimates expect this prevalence to increase further in the coming years. Decades of research have revealed environmental, genetic, physiological, and behavioral influences in the manifestation and progression of obesity, but the mechanisms underpinning these interactions remain poorly understood. Diet's impacts on gut health and obesity cannot be understated. Recent research suggests that what we eat can modulate our weight and determine the composition and structure of the microorganisms inhabiting our guts. Kombucha, a fermented beverage produced when a Symbiotic Culture of Bacteria and Yeast (SCOBY) is grown on tea infusions, has received increasing scientific and media attention given its dietary health benefits. Unfortunately, no studies have yet confirmed these findings in humans. The present study aims to address this knowledge gap by investigating the long-term (eight weeks) impacts of consistent black-tea kombucha consumption on gut microbial health and assessing how these impacts differ between participants with and without obesity. The study measured gut microbiota changes, short-chain fatty acid (SCFA) content, and intestinal permeability of all participants at study enrolment and intervention termination. It's important to note that this study used a pre-post intervention design without a parallel control group that did not consume kombucha. Prospective participants were recruited through institutional emails and social media advertisements and were required to undergo questionnaire-based screening before study enrolment. Excluded were those who consumed kombucha (at least once a week over the past month); regularly used anti-inflammatory drugs, corticoids, or drugs that affect lipid or glucose metabolism; used antioxidant or vitamin supplements; took antibiotics 3 months before the study; had infectious or allergic episodes in the last month; were on weight loss diets; did not present a stable weight over the 3 months before the study (±3 kg); had an aversion to kombucha; had alcohol intake greater than 105 g of ethanol/week for women and 210 g for men; smokers; and pregnant and lactating women. All participants were subjected to a baseline evaluation, which included collecting demographic data, medical records, laboratory examinations, and biological samples (blood, stools, and urine). Simultaneous food frequency questionnaires (FFQs) and the International Physical Activity Questionnaire (IPAQ) were maintained to ensure that participants did not significantly alter their routine diets and physical activity levels. Researchers used a combination of titration, high-performance liquid chromatography (HPLC), ultra-performance liquid chromatography mass spectrometry (UPLC-MS), and Folin–Ciocalteu colorimetric assays to characterize the composition of their black-tea kombucha. Microbiological characterization (serial dilution approach), intestinal permeability (lactulose and mannitol), and biomarker identification (ELISAs) helped elucidate the impacts of consistent kombucha consumption on the gut. Laboratory-generated black-tea kombucha presented with a low pH (increased acidity) and a residual slightly sweet taste (sugar added for fermentation). After 8 weeks of kombucha consumption, physical activity patterns remained mostly unchanged, with only 3 participants showing a shift, 2 in the normal weight group and 1 in the obese group. UPLC-MS-based characterization of the kombucha revealed it to be a rich source of phenolic compounds (n = 145), including flavonoids (81%) and phenolic acids (19%). Frequent kombucha consumption was found to promote the growth of commensal Bacteroidota and Akkermanciaceae bacteria, with bacterial growth strongest in obese participants. Obese participants also benefited from an increase in Subdoligranulum (butyrate producer) populations and significant reductions in Ruminococcus and Dorea (obesity-associated) genera. Encouragingly, Ruminococcus and Dorea levels, which were significantly higher in the obese cohort compared to normal-weight participants, were reduced in the obese participants to normal baseline levels by intervention termination. However, the study did not find significant differences in short-chain fatty acid (SCFA) concentrations (acetate, butyrate, and propionate) in the stool samples of participants in either group, before or after the 8-week kombucha consumption. Similarly, markers of intestinal permeability, such as the lactulose to mannitol ratio in urine and plasma zonulin concentrations, did not show significant changes between or within groups after the intervention. Relative abundance of major phyla (top 10) (A), families (top 10) (B), and genera (top 10) (C) in normal weight and obese groups at baseline (T0) and after 8 wk of regular consumption of black tea kombucha (T8). The present study establishes the beneficial gut microbial effects of frequent kombucha consumption, particularly in obese or overweight individuals who experience greater benefits than their normal-weight counterparts in terms of bacterial modulation. While the intervention led to these positive shifts in gut bacteria and fungal composition, it's noteworthy that direct measures of short-chain fatty acid production in stool and markers of intestinal permeability did not significantly change within the 8-week study period. Future research with a control group and potentially longer intervention durations would be beneficial to confirm these findings and explore potential clinical impacts. Regular kombucha consumption positively influenced gut microbiota in both normal and obese groups, with more pronounced effects in the obese group, suggesting that it may be especially beneficial for those individuals. Hugo Francisco de Souza is a scientific writer based in Bangalore, Karnataka, India. His academic passions lie in biogeography, evolutionary biology, and herpetology. He is currently pursuing his Ph.D. from the Centre for Ecological Sciences, Indian Institute of Science, where he studies the origins, dispersal, and speciation of wetland-associated snakes. Hugo has received, amongst others, the DST-INSPIRE fellowship for his doctoral research and the Gold Medal from Pondicherry University for academic excellence during his Masters. His research has been published in high-impact peer-reviewed journals, including PLOS Neglected Tropical Diseases and Systematic Biology. When not working or writing, Hugo can be found consuming copious amounts of anime and manga, composing and making music with his bass guitar, shredding trails on his MTB, playing video games (he prefers the term ‘gaming'), or tinkering with all things tech. Please use one of the following formats to cite this article in your essay, paper or report: Black tea kombucha reduces harmful gut microbes linked to obesity. "Black tea kombucha reduces harmful gut microbes linked to obesity". "Black tea kombucha reduces harmful gut microbes linked to obesity". Black tea kombucha reduces harmful gut microbes linked to obesity. Learn how experts are advancing benzodiazepine analysis and detection using insights from the lab. News-Medical.Net provides this medical information service in accordance with these terms and conditions. Please note that medical information found on this website is designed to support, not to replace the relationship between patient and physician/doctor and the medical advice they may provide. Hi, I'm Azthena, you can trust me to find commercial scientific answers from News-Medical.net. Registered members can chat with Azthena, request quotations, download pdf's, brochures and subscribe to our related newsletter content. A few things you need to know before we start. While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles. Please do not ask questions that use sensitive or confidential information.

How cancer patients relying on daratumumab feel physically before starting the therapy can predict how long they will live and how well they will respond to the anti- multiple myeloma (MM) drug, according to a new study published in the European Journal of Haematology. The study analyzed data from 1,804 patients enrolled in three large-scale clinical trials-MAIA, POLLUX, and CASTOR. Across these trials, the median patient age was 66 years, and 44% were female. About half of the patients were randomly assigned to receive daratumumab-containing therapy, while the other half received standard treatments without daratumumab. The responses revealed that patients with lower scores benefited more from daratumumab, lived longer, and had a lower risk of disease progression. This is the first study to demonstrate that patient-reported physical function at treatment initiation can predict which patients derive the greatest survival benefit from daratumumab, a monoclonal antibody widely used in the treatment of multiple myeloma." The study offers a simple, low-cost way to improve treatment decisions, particularly for older or frail patients. "How well cancer patients can perform daily tasks like walking or getting dressed-is a powerful predictor of survival outcomes and treatment benefit in people with multiple myeloma receiving daratumumab-based therapies." In this low physical function group, daratumumab reduced the risk of death from any cause by 47% (hazard ratio 0.53 [95% CI: 0.40–0.70], P < 0.001) and the risk of cancer progression by 66% (hazard ratio 0.34 [0.22–0.53], P < 0.001) compared to those not receiving daratumumab. In contrast, patients who reported being physically well before treatment-the high physical function group-had less benefit. Their risk of death was reduced by only 14%, which was not statistically significant (HR 0.86 [0.62–1.19], P = 0.364) compared to those not receiving daratumumab. They did, however, experience a 47% reduction in the risk of cancer progression (HR 0.53 [0.42–0.67], P = 0.034), showing that while daratumumab was still effective in this group, the magnitude of benefit was smaller (47%) than that seen in patients with low physical function (66%). These results held true regardless of the patient's age, sex, weight, cancer stage, doctor-assessed health score (ECOG), or number of other health problems. Interestingly, the commonly used doctor rating of a patient's general health (ECOG) did not help predict who would benefit more - but the patients' own reports of their physical function did. "Physical function is a predictive and prognostic marker that complements ECOG-PS, supporting its use in informing therapy decisions for daratumumab-based treatments," the authors write. Doctors often use the ECOG Performance Status – a scale from 0 (fully active) to 5 (dead) – to determine how well a patient is functioning. But the study, according to Dr. Abuhelwa, found that many patients classified as "fully active" by ECOG reported significant physical challenges themselves. This highlights a critical gap-ECOG alone may not capture the full picture. The authors draw two major conclusions from their extensive analysis. In the first, they found that patients who reported lower physical function at treatment initiation received the greatest survival benefit from daratumumab. "These findings were independent of traditional doctor-assessed tools, such as ECOG performance status. Importantly, daratumumab did not lead to more serious side effects in patients with low physical function," Dr. Abuhelwa points out. "Bottom line: What patients say about their physical limitations-right at the start of therapy-provides critical, actionable insights to guide cancer treatment decisions." In 2022, there were an estimated 188,000 new cases and 121,000 deaths worldwide. On the importance of the study, co-author Dr. Ashley Hopkins, Associate Professor at Flinders University in Australia, said, "This is a timely and significant contribution. It highlights how patient-centered data can meaningfully guide complex treatment decisions in oncology. The study serves as a strong reminder to health professionals to seriously consider what cancer patients say about their physical function before initiating treatment." Prof. Humaid Al-Shamsi, a co-author from the UAE's Burjeel Cancer Institute, added, "This study highlights the growing importance of patient-centered care in oncology. By listening closely to how patients feel at the start of treatment, we can better personalize therapies and improve outcomes – especially for those who are older or more physically vulnerable. It's a step forward in making cancer care more precise, compassionate, and effective." The authors suggest their study could have wide-reaching implications. In it, they call on clinicians to incorporate patient-reported physical function into treatment planning; urge policymakers to promote the use of patient-reported outcomes (PROs) in clinical trials and routine care; and encourage drug developers to consider PROs when designing future cancer studies. However, they maintained that further research and prospective studies were warranted to confirm the identified treatment benefits and explore whether they would extend to other contemporary multiple myeloma treatment regimens. Said Dr. Abuhelwa, "With further validation, patient-reported outcomes could become an essential part of personalized treatment strategies, ultimately improving both survival and quality of life for people living with multiple myeloma." Learn how experts are advancing benzodiazepine analysis and detection using insights from the lab. News-Medical.Net provides this medical information service in accordance with these terms and conditions. Please note that medical information found on this website is designed to support, not to replace the relationship between patient and physician/doctor and the medical advice they may provide. Hi, I'm Azthena, you can trust me to find commercial scientific answers from News-Medical.net. Registered members can chat with Azthena, request quotations, download pdf's, brochures and subscribe to our related newsletter content. A few things you need to know before we start. While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles. Please do not ask questions that use sensitive or confidential information.

To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued an Evidence in Focus article, published May 14, 2025, online in Neurology®. AAN Evidence in Focus articles highlight the strength of the current evidence for new therapies for neurological conditions. This article reviews evidence available for the efficacy and safety of delandistrogene moxeparvovec. Duchenne muscular dystrophy, which primarily affects those of male sex, is an inherited genetic condition that causes muscle weakness. People with this disease are missing a protein called dystrophin that helps maintain muscle health. Delandistrogene moxeparvovec is a one-time gene therapy infusion approved by the Food and Drug Administration in June 2024 for use in those age four and older. "While the prognosis for individuals with Duchenne muscular dystrophy has improved in recent decades thanks to corticosteroids and supportive care, significant functional limitations persist for those affected by the condition," said author Maryam Oskoui, MD, of McGill University in Quebec, Canada, and a Fellow of the American Academy of Neurology. "Recent advances in genetic therapies for this disease have brought hope to many, with several gene replacement therapies currently being evaluated in clinical trials, and one-delandistrogene moxeparvovec-already approved by the FDA. This Evidence in Focus article offers a timely and objective overview of the existing data and explores key clinical considerations surrounding the use of delandistrogene moxeparvovec. However, it is important to acknowledge that evidence supporting its potential effectiveness and safety remains limited." The Evidence in Focus article states that both Class I studies of delandistrogene moxeparvovec failed to meet their primary outcome measures related to motor function, a person's ability to control movements. Research showed the therapy may possibly slow the decline of other measures of motor function by a small amount. However, the article states it is difficult to know how much of this benefit is due to the gene therapy and how much may additionally be influenced by the high dose steroid medications that are taken with the therapy. Based on the current research, it has yet to be determined whether the therapy will extend a person's life or improve their quality of life. The therapy does not cure Duchenne muscular dystrophy. Those considering this gene therapy should ask their doctor if insurance will pay for the treatment. Oskoui, M., et al. (2025) Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus Report of the AAN Guidelines Subcommittee. In this interview, industry expert Dr. Lohit Khera discusses the evolving role of microRNA in research, diagnostics, and precision medicine, highlighting the latest innovations in RNA extraction and analysis Learn how experts are advancing benzodiazepine analysis and detection using insights from the lab. News-Medical.Net provides this medical information service in accordance with these terms and conditions. Please note that medical information found on this website is designed to support, not to replace the relationship between patient and physician/doctor and the medical advice they may provide. Hi, I'm Azthena, you can trust me to find commercial scientific answers from News-Medical.net. Registered members can chat with Azthena, request quotations, download pdf's, brochures and subscribe to our related newsletter content. A few things you need to know before we start. Please check the box above to proceed. While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles. Please do not ask questions that use sensitive or confidential information.

While it may seem intuitive that people would die without life-saving medications, Penn and Harvard researchers have connected losing a federally funded prescription drug assistance program and an increase in mortality. The program, called the Medicare Part D Low-Income Subsidy (LIS), helps 14.2 million low-income Medicare beneficiaries, many of whom are older Americans, afford their medications. Nationally, 12.5 million people who are eligible for and enrolled in both Medicare and Medicaid ("dual eligibles") automatically qualify for the LIS, which is worth about $6,200/year. Chan School of Public Health, reveals that losing Medicaid coverage-and with it, the LIS-was associated with significant increases in mortality among low-income Medicare beneficiaries. When Medicare beneficiaries lose Medicaid, which happens to more than 900,000 people each year, they also risk losing the LIS and therefore, being able to afford the medicines they need." The study included nearly 1 million low-income Medicare beneficiaries whose Medicaid coverage ended. The researchers compared two groups: individuals who lost Medicaid from January to June who were removed from LIS by the following January (7–12 months later), and those who lost Medicaid from July to December lost LIS the next January (13–18 months later). This allowed the team to compare mortality rates during the period when some had lost LIS and others had not. Mortality was 4 percent higher among individuals who lost the LIS earlier than those who retained it for a longer period. For example, mortality was 22 percent higher among people using HIV antiretroviral therapy. The researchers found that over one half of individuals who lost Medicaid regained it within one year, suggesting that many were dropped from Medicaid despite remaining eligible. "These findings show that helping low-income Medicare beneficiaries who are eligible for Medicaid stay enrolled and retain the LIS can save lives since it preserves access to essential medications," said senior author José F. Figueroa, MD, MPH, an associate professor of health policy and management at Harvard Chan School. The authors note that recent increases in Medicaid coverage losses among older adults raise concerns about potential losses of the LIS. Added Roberts, "As policymakers consider major changes to the Medicaid program, preserving Medicaid coverage for older adults is critical to ensuring that they keep the LIS." Support for this work comes from the National Institute on Aging (R01AG076437; R01AG081151; RF1AG088640; T32AG000037), the Agency for Healthcare Research and Quality (R01HS029453), and Arnold Venture. Roberts, E. T., et al. (2025) Loss of Subsidized Drug Coverage and Mortality among Medicare Beneficiaries. Learn how experts are advancing benzodiazepine analysis and detection using insights from the lab. News-Medical.Net provides this medical information service in accordance with these terms and conditions. Please note that medical information found on this website is designed to support, not to replace the relationship between patient and physician/doctor and the medical advice they may provide. Hi, I'm Azthena, you can trust me to find commercial scientific answers from News-Medical.net. Registered members can chat with Azthena, request quotations, download pdf's, brochures and subscribe to our related newsletter content. A few things you need to know before we start. While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles. Please do not ask questions that use sensitive or confidential information.