Adolescents and young adults with Duchenne (DMD) or Becker muscular dystrophy (BMD) who have more siblings typically report being better prepared to transition to adult life, a new study has found. Findings show people with DMD or BMD had the most difficulty with activities of daily living, education,…
News
Five people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakness affecting the face and throat — have thus far been treated with the gene therapy candidate BB-301 in an ongoing clinical trial, treatment developer Benitec Biopharma said in a…
A Phase 1 clinical trial testing SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD), is now done enrolling healthy volunteers, its developer Satellos Bioscience announced. “The completion of healthy volunteer enrollment in our Phase 1 trial marks a significant milestone in our mission to develop transformative…
A fundraiser through the online role-playing game World of Warcraft has raised more than $2 million to support CureDuchenne, an organization dedicated to combating Duchenne muscular dystrophy (DMD). The fundraiser, which launched last year in partnership with Warcraft’s maker Blizzard Entertainment, is the largest contribution from a…
Treatment with oral therapy ifetroban led to improvements in measures of heart function in people with Duchenne muscular dystrophy (DMD), according to top-line results from a clinical trial. The “impressive results” are a “pivotal moment” for developer Cumberland Pharmaceuticals, “and, more importantly, for the DMD community,” A.J. Kazimi,…
Entrada Therapeutics announced it has been cleared in the U.K. to start a Phase 1/2 clinical trial of ENTR-601-44, at increasing doses, in Duchenne muscular dystrophy (DMD) patients with a mutation in the DMD gene amenable to exon 44 skipping. With this decision by U.K.’s Medicines and…
The Muscular Dystrophy Association (MDA) has announced its 2025 Legacy Awards, and will honor two individuals who have made pioneering strides toward improving life for people affected by muscular dystrophy. The 2025 MDA Legacy Award for Achievement in Clinical Research will be given to Katherine Mathews, MD, a…
Two years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl), motor function continues to improve in boys with Duchenne muscular dystrophy (DMD) who entered a global clinical trial able to walk. That’s according to top-line findings from part two of the Phase 3 EMBARK trial (NCT05096221), which…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to DYNE-101, a treatment candidate for myotonic dystrophy type 1 (DM1) — in which disease symptoms begin during adulthood — that’s now being tested in a clinical trial involving DM1 patients. This status is awarded by the…
Once a week treatment with a corticosteroid was seen to lower some biomarkers of muscle damage and inflammation, helping to improve muscle function in people with some forms of muscular dystrophy, according to a pilot study in the U.S. Prednisone was given once weekly for six months to 19…
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