News

Epic Bio’s EPI-321 named FDA orphan drug for FSHD

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,…

Satellos nominates SAT-3247 as lead treatment candidate for DMD

Satellos Bioscience is hoping to bring SAT-3247, its newly nominated lead treatment candidate for Duchenne muscular dystrophy (DMD), into clinical trials next year. The company announced that it’s working to establish a good manufacturing practice (GMP) setup to make the medication, and is in the midst of experiments…

Harley-Davidson dealers raise over $650K at 36th MDA Ride for Life

Continuing years of support for the Muscular Dystrophy Association (MDA), the Eastern Pennsylvania Harley-Davidson Dealers Association (EHDDA) just raised nearly $658,000 at the 36th Annual MDA Ride for Life. Proceeds from the event, held in October this year, will help further the organization’s mission to empower those with…

Sarepta planning to seek expanded Elevidys approval after EMBARK

While a Phase 3 clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) has failed to meet its main goal, the results nonetheless showed that, compared with a placebo, the approved gene therapy led to notable improvements across numerous measures of physical function in boys with Duchenne muscular dystrophy (DMD). Top-line findings…

DYNE-251 for DMD, DYNE-101 for DM1 showing safety in early trials

DYNE-251, an experimental treatment for Duchenne muscular dystrophy (DMD), is being well tolerated by patients in an ongoing clinical trial, according to the therapy’s developer, Dyne Therapeutics. Dyne also reported positive safety data from an ongoing trial of DYNE-101, the company’s investigational therapy for myotonic dystrophy type…

In models of DMD, gene editing allows exon skipping

A gene editing approach can restore dystrophin production in cell and animal models of Duchenne muscular dystrophy (DMD), according to its researchers. The study, “Targeting Duchenne Muscular Dystrophy by Skipping DMD Exon 45 with Base Editors,” was published in Molecular Therapy Nucleic Acids. DMD is caused by…