The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting their growth and with fewer side effects compared to standard corticosteroids, according to 18-month data from a Phase 2 trial and its extension study. Findings were reported in the study, “…
In a Facebook Live event hosted by the Muscular Dystrophy Association (MDA), experts discussed the effect that the COVID-19 pandemic has had on research efforts in muscular dystrophy, as well as the ongoing development of a potential COVID-19 vaccine. Clinical trials are a critical…
Real-world and natural history data could be used to supplement, or perhaps replace, the use of a placebo in future clinical trials in people with Duchenne muscular dystrophy (DMD), a new study suggests. The study, “Suitability of external controls for drug evaluation in Duchenne muscular dystrophy,”…
Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) that clarified regulatory requirements to start the company’s next clinical trial of SRP-9001, an investigational gene therapy for Duchenne muscular dystrophy (DMD). During the type C “written response only” meeting, the…
An imbalance in cells producing proteins called aldehyde dehydrogenases (ALDHs) — favoring fat formation in tissue — is evident in the muscles of people with Duchenne muscular dystrophy (DMD), a study reports. A better understanding of these cells could open new avenues of treatment, its researchers said. The…
Santhera Pharmaceuticals has obtained the worldwide rights to vamorolone (VBP15), an experimental therapy for Duchenne muscular dystrophy (DMD), the company announced. The company has reached an agreement with ReveraGen Biopharma, the original developer of vamorolone, and Actelion Pharmaceuticals’ spin-off Idorsia, which had acquired an…
September is Duchenne Action Month, an awareness, fund-raising, and educational campaign supporting people with Duchenne muscular dystrophy (DMD) Hosted by Parent Project Muscular Dystrophy (PPMD), each week of the month is organized around a different theme, with scheduled events, as well as various resources and social media…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
This year, the Muscular Dystrophy Association (MDA) will celebrate MDA National Muscular Dystrophy Awareness Month, held every September, with a series of online events. The events will be held virtually for the first time to ensure maximum participation, while promoting social distancing and other safety measures for people with…
An application requesting the approval of casimersen (SRP-4045) to treat Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping has been accepted and placed under priority review by the U.S. Food and Drug Administration (FDA), Sarepta Therapeutics announced. A regulatory decision is expected no later than Feb. 25, 2021. The…
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