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The Muscular Dystrophy Association (MDA) has announced the agenda for its 2026 MDA Clinical & Scientific Conference — slated for March 8-11 in Orlando, Florida — and the focus, the nonprofit says, is on “groundbreaking research and clinical achievements.” The annual event, held in a different locale each…

Note: This story was updated Oct. 20, 2025, to correct the reported increase in dystrophin levels observed at higher doses of brogidirsen in the Phase 1/2 study. Brogidirsen (NS-089/NCNP-02), NS Pharma’s investigational therapy for people with Duchenne muscular dystrophy (DMD) who are amenable to exon 44 skipping, has…

Three boys with Duchenne muscular dystrophy (DMD) who were given the experimental gene therapy GNT0004 in a clinical trial were seeing sustained improvements in motor function and reductions in markers of muscle damage two years after the one-time treatment. Genethon, GNT0004’s developer, presented the findings at the European…

Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older, making Agamree the first approved DMD treatment in Canada. Canada’s approval is the sixth for Agamree to treat DMD. The therapy is approved in the U.S., the U.K.,…

Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences, is demonstrating the potential to reverse disease progression in boys and young men with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, or DMD44. That’s according to one-year data from the Phase 1/2 EXPLORE44 trial (NCT05670730)…

For boys who start corticosteroids to treat Duchenne muscular dystrophy (DMD), being taller is linked to slower growth, while being older is linked to more weight gain as they move into adolescence and near the loss of their ability to walk, according to Phase 3 clinical data. This implies…

Satellos Bioscience asked the U.S. Food and Drug Administration (FDA) for clearance to conduct a Phase 2 clinical trial testing SAT-3247 in children with Duchenne muscular dystrophy (DMD) who are able to walk. The three-month study will randomly assign participants to SAT-324, taken as a pill, or a placebo.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CRD-003, an investigational gene therapy for limb-girdle muscular dystrophy type 2i/R9 (LGMDR9). The FDA gives this designation to experimental therapies designed to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S.

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to NS-051/NCNP-04, an exon-skipping therapy designed to treat certain people with Duchenne muscular dystrophy (DMD). The FDA gives this designation to experimental treatments designed to improve care for rare disorders, specifically conditions affecting fewer than 200,000 people…

A Phase 1/2 clinical trial testing SAR446268, an experimental gene therapy for myotonic dystrophy type 1 (DM1), is enrolling patients. The trial, dubbed BrAAVe (NCT06844214), is expected to enroll approximately 32 DM1 patients aged 10-50. The study is actively recruiting participants at sites in Florida, New York,…