Early Trial Data Highlights Potential of Gene Therapy GNT0004 for Duchenne Muscular Dystrophy
March 18th 2025Long-term follow-up data from an early-phase study of an AAV8-based gene therapy for DMD suggest the treatment was well-tolerated and provided sustained biochemical and functional benefits.
Mechanism and Promise Behind Del-Zota for Duchenne Muscular Dystrophy: Michael Flanagan, PhD
March 18th 2025The chief scientific officer at Avidity Biosciences provided clinical perspective on the function and mechanism of del-zota, an investigational antisense treatment in development for DMD amenable to exon 44 skipping. [WATCH TIME: 3 minutes]
Eteplirsen Treatment Significantly Slows Heart Function Decline in Duchenne Muscular Dystrophy
March 17th 2025A recent study shows that eteplirsen, a drug promoting dystrophin production, significantly slowed the decline in heart function, specifically left ventricular ejection fraction, in patients with Duchenne muscular dystrophy.
The Health and Productivity Impacts of Time Changes in Different Populations
March 16th 2025Experts discussed the negative health and societal effects of daylight saving time, advocating for a permanent switch to standard time to improve sleep, well-being, and productivity. [WATCH TIME: 6 minutes]
Vulnerable Populations and the Impact of Time Changes on Sleep Disruption
March 15th 2025Experts discussed the biological misalignment caused by daylight saving time, emphasizing its negative effects on sleep, particularly in adolescents, and advocating for a shift to standard time to better align with natural circadian rhythms. [WATCH TIME: 6 minutes]
World Sleep Day with Dr. Eric Olson: Advancing Sleep Health and Awareness
March 14th 2025On World Sleep Day, the president of the American Academy of Sleep Medicine discussed the importance of sleep for health, the risks of sleep deprivation, nonpharmacologic strategies to improve sleep, and the need for greater awareness of sleep disorders.
Rethinking Rest on World Sleep Day: Neurologist’s Take on Sleep Priorities
March 14th 2025Anne Marie Morse, DO, director of Child Neurology and Pediatric Sleep Medicine at Geisinger Janet Weis Children’s Hospital, dove into the importance of sleep health, its impact on brain function, and emerging research in honor of World Sleep Day.
What to Expect From the 2025 CMSC Annual Meeting: Kathleen Costello, CRNP, MSCN
March 13th 2025The interim chief executive officer at the Consortium of Multiple Sclerosis Centers invites healthcare professionals to the Consortium’s annual meeting from May 28–31 in Phoenix, Arizona. [WATCH TIME: 2 minutes]
Misdiagnosed Migraine Leads to Significant Burden on Healthcare Resource Utilization
March 13th 2025New research highlights the significant economic burden of misdiagnosed migraine patients, revealing higher healthcare costs and resource utilization over time compared to those with accurate diagnoses.
Neflamapimod Shows Promising Phase 2b Extension Data in Dementia With Lewy Bodies
Published: March 12th 2025 | Updated: March 13th 2025Participants who received the new batch of capsules during the extension phase experienced a lower incidence of falls compared with those taking the old capsules or placebo.