Marisa Wexler, MS,  senior science writer—

Note: This story was updated Sept. 10, 2025, to correct the secondary headline from Columbia University to the University of Iowa. Columbia University awarded the 2025 Louisa Gross Horwitz Prize to three scientists whose research has helped lay the groundwork to develop new treatments for Duchenne muscular dystrophy (DMD). This…

Researchers have developed a way to enhance the efficacy of exon-skipping therapies for Duchenne muscular dystrophy (DMD), and the approach showed promise in a mouse model of the disease. The study, “A Combinatorial Oligonucleotide Therapy to Improve Dystrophin Restoration and Dystrophin-Deficient Muscle Health,” was published in Molecular Therapy…

Preliminary data are expected this year for a Phase 1/2 clinical trial testing the investigational therapy SRP-1003 in people with myotonic dystrophy type 1 (DM1), according to an announcement from Sarepta Therapeutics. The trial recently hit one of two prespecified enrollment targets, prompting a review of safety data.

The steroid therapy Agamree (vamorolone), which is used for helping to preserve muscle function in people with Duchenne muscular dystrophy (DMD), will be sold and promoted in Turkey by Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. (GEN), per an agreement with Santhera Pharmaceuticals. “As leaders…

A protein called MYOD, which is known to control the growth of muscle stem cells during muscle repair, has long been known to be able to turn on genes, but a recent study has now found that MYOD can also turn off certain genes. According to researchers, the findings may…

Enrollment is now ongoing in a first-in-human trial testing EPI-321, an epigenetic therapy designed to address the root cause of facioscapulohumeral muscular dystrophy (FSHD), a disease type in which symptoms like muscle weakness typically become apparent by adulthood. The Phase 1/2 clinical trial (NCT06907875) is expected to enroll…

Note: This story was updated July 30, 2025, to include data from part two of the EMBARK trial. Sarepta Therapeutics will soon resume shipping Elevidys (delandistrogene moxeparvovec-rokl) for ambulatory Duchenne muscular dystrophy (DMD) patients, following a review of safety data by the U.S. Food and Drug Administration (FDA),…

Sarepta Therapeutics is voluntarily halting all shipments of Elevidys (delandistrogene moxeparvovec-rokl) in the U.S. after a request from the U.S. Food and Drug Administration (FDA). The company had initially refused to pause the shipments of its gene therapy for Duchenne muscular dystrophy (DMD) for individuals with DMD who…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Precision Biosciences. The designation provides companies with extra incentives to develop experimental therapies to treat rare diseases that mainly affect…

The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting next month to review the data on deramiocel, an experimental cell therapy up for approval to treat heart disease in people with Duchenne muscular dystrophy (DMD). The FDA has informed Capricor Therapeutics…