Andrea Lobo,  —

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease status to SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) developed by Solid Biosciences. The designation is given to therapies with the potential to prevent or treat rare diseases that primarily affect children and…

Long-term treatment with AOC 1001 continues to be safe and may reverse the progression of myotonic dystrophy type 1 (DM1), according to new data. A year of treatment eased myotonia, that is, the inability of muscle to relax after they’ve contracted, and improved muscle strength along with outcomes…

SAT-3247, an oral therapy candidate for rebuilding muscle tissue, improved muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD). Developed by Satellos Bioscience, the therapy is the lead treatment candidate for Duchenne muscular dystrophy (DMD), with the first clinical trial of SAT-3247 for DMD…

Epic Bio’s epigenetic therapy EPI-321 has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA) as a treatment for the most common form of muscular dystrophy in adults, called facioscapulohumeral muscular dystrophy (FSHD). The designation is given to medications that could prevent,…

Fulcrum Therapeutics has completed patient enrollment for its Phase 3 clinical trial evaluating losmapimod as a potential treatment for people with Facioscapulohumeral muscular dystrophy (FSHD). The study, dubbed REACH (NCT05397470), is evaluating the efficacy and safety of losmapimod against a placebo in FSHD adult patients across…

More than 420 “Fill the Boot” events will raise money throughout Labor Day weekend to advance research and care for muscular dystrophy. The campaign results from a partnership between the Muscular Dystrophy Association (MDA) and the International Association of Fire Fighters (IAFF). The firefighters go to…

The U.S. Food and Drug Administration (FDA) has accepted and given priority review to Italfarmaco Group’s application requesting that oral givinostat be approved to treat Duchenne muscular dystrophy (DMD). Priority review, which shortens the consideration period to six months from 10, is given to therapies with…

The U.S. Food and Drug Administration (FDA) has cleared Benitec Biopharma to start a Phase 1b/2a clinical trial of the investigational gene therapy BB-301 for swallowing difficulties associated with oculopharyngeal muscular dystrophy (OPMD). Dosing is expected to start later this year, following the rollover of participants in…

Stem cells derived from the amniotic membrane of pregnant women after childbirth “could provide therapeutic benefits” for people with Duchenne muscular dystrophy (DMD), according to researchers in Japan. These stem cells, known as human mesenchymal stromal cells, were able to delay DMD progression and preserve muscle function in a…

Sarepta Therapeutics is calling for applications for the 6th annual Route 79, The Duchenne Scholarship Program, for the 2023-2024 academic year. The scholarships, of up to $5,000, will be awarded to up to 20 students living with Duchenne muscular dystrophy (DMD), and up to five siblings of…