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.
News
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…
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.,…
Recent Posts
- The rule of 10 helps me navigate bad days with LGMD
- I have 5 essential tools for managing cold and flu season with Duchenne MD
- FDA fast tracks Dyscorban for treating heart problems in Duchenne
- My emotions blurred as I watched my play from ‘The Other Side’
- MDA-led project to map how human muscles repair themselves