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Note: This story was updated Nov. 5, 2025, to clarify that enrollment in the follow-up study is ongoing. Dosing has begun in a long-term follow-up study testing Satellos Bioscience’s experimental oral therapy SAT-3247 in men with Duchenne muscular dystrophy (DMD). The study — called LT-001 (NCT06867107) —…

In a deal worth roughly $12 billion, pharmaceutical giant Novartis has agreed to acquire Avidity Biosciences, to develop several promising experimental therapies for various types of muscular dystrophy. The core of the acquisition is Avidity’s antibody-oligonucleotide conjugate (AOC) platform. This innovative technology attaches a small piece of…

Use of the oral therapy BBP-418 appeared to improve walking ability and lung function in people with limb-girdle muscular dystrophy type 2i (LGMD2i) — characterized by muscle wasting at the shoulders and the hips — in a Phase 3 clinical trial, according to new interim data announced by developer…

Data from two clinical trials confirm that Agamree (vamorolone), an approved anti-inflammatory treatment for people with Duchenne muscular dystrophy (DMD), blocks the action of a receptor involved in regulating the balance of salt and water in the body, and may have advantages over traditional corticosteroids. A study found…

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…