Continuing corticosteroid treatment into adulthood was associated with slower lung-function decline in people with Duchenne muscular dystrophy (DMD), according to a new study. “The results of this study provide further evidence to support continuing [corticosteroid] treatment into late adolescence and adulthood” for people with DMD,…
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The Muscular Dystrophy Association (MDA) brought its Engage Community Seminar to Hershey, Pennsylvania, on July 18, uniting individuals with neuromuscular diseases, caregivers, and medical experts for a day of education and connection. Hosted in collaboration with the Penn State Health Milton S. Hershey Medical Center, the one-day event featured a…
The U.S. Food and Drug Administration (FDA) is considering whether to conditionally approve zeleciment rostudirsen (z-rostudirsen), an experimental therapy for Duchenne muscular dystrophy (DMD) in patients with mutations that are amenable to exon 51 skipping. Z-rostudirsen developer Dyne Therapeutics announced that the FDA has agreed to review an…
Commendamide, a metabolite produced by beneficial gut bacteria that are depleted in people with Duchenne muscular dystrophy (DMD), protects muscle cells from damage by enhancing antioxidant mechanisms, a study found. “These findings provide new insights into the gut-muscle axis in DMD” and “support further investigation of microbiota-derived metabolites as…
CureDuchenne, a U.S.-based nonprofit that funds research into new treatments for Duchenne muscular dystrophy (DMD), has launched a national public service announcement campaign to highlight the urgency behind research for people with DMD. The new advocacy campaign, dubbed A Cure Can’t Wait, highlights…
Two major steps are now complete in an early clinical trial testing a one-time epigenetic therapy — a treatment designed to turn on or off a specific gene without changing the underlying DNA — for facioscapulohumeral muscular dystrophy (FSHD), a genetic disease that characteristically affects the muscles of the…
This year’s Be Their Muscle campaign has raised more than $641,000 to support MDA Summer Camp for children and adolescents with muscular dystrophy and other neuromuscular diseases, and to help advance research and care for these patient populations. Resulting from a partnership between the Muscular Dystrophy Association (MDA)…
The U.S. Food and Drug Administration (FDA) is expected to decide by Feb. 28, 2027, whether to grant traditional approval to Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (DMD). Both medications are currently approved in…
Amid its ongoing review of the cell therapy deramiocel for people with Duchenne muscular dystrophy (DMD), the U.S. Food and Drug Administration (FDA) has scheduled a meeting with Capricor Therapeutics, its developer, to discuss the company’s resubmission of an application…
The Department of Justice (DOJ) has released a memorandum arguing that states are not required to provide in-home or community-based care to people with disabilities, including those with muscular dystrophy (MD). The move drew sharp condemnation from the Muscular Dystrophy Association (MDA), which stated in a press…
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