A Phase 1/2 clinical trial testing SRP-1003, a treatment for myotonic dystrophy type 1 (DM1), is progressing as planned and continues to enroll patients at higher doses. The Phase 1/2 study (NCT06138743) is expected to enroll 78 adults, ages 18 to 65, who have a genetically confirmed diagnosis…
New findings from research in mice are challenging longstanding beliefs about the causes of Duchenne muscular dystrophy (DMD), with evidence showing that the genetic disease is marked by abnormalities in muscle stem cells during fetal development — indicating DMD may start in the womb. According to the researchers, these findings suggest…
The U.S. Food and Drug Administration (FDA) has approved an update to the prescribing information for Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec-rokl), removing its conditional approval for DMD patients who can’t walk and including new safety warnings and precautions in light of two deaths related to the…
Agamree (vamorolone), a type of corticosteroid approved to help preserve muscle function in people with Duchenne muscular dystrophy (DMD), appears to work as well as traditional corticosteroids at maintaining walking ability, but carries a lower risk of side effects such as broken bones, eye problems, and stunted growth.
Exon-skipping therapies Amondys 45 (casimersen) and Vyondys 53 (golodirsen) show “positive and encouraging trends” for people with Duchenne muscular dystrophy (DMD), even though top-line results from a Phase 3 trial testing the treatments showed the study failed to meet its main goal, developer Sarepta Therapeutics said. Results from…
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…
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