News

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to Edgewise Therapeutics‘ EDG-5506 as a potential treatment for Duchenne muscular dystrophy (DMD). EDG-5506 also was awarded FDA orphan drug status for Becker muscular dystrophy (BMD) as a possible…

Growth hormone (GH) or testosterone can better protect against spinal fractures than a common osteoporosis medication alone in boys with Duchenne muscular dystrophy (DMD) or severe Becker muscular dystrophy (BMD), according to a long-term study. Used to prevent or lessen osteoporosis, or weak and brittle bones, zoledronic acid (ZA) is…

NS Pharma has opened a program for people with Duchenne muscular dystrophy (DMD), and their caregivers and family members, to share insights that might help others living with this most common form of muscular dystrophy. Called Duchenne Heroes, it relates the experiences of patients and…

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 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…

The expression of a protein called ACTC1 in muscle fibers is critical for muscle repair — and vital to the survival of transplanted human muscle stem cells — a new study shows. These findings may have important implications for the development of cell therapies for muscular dystrophy and other…

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…

The blood of boys with Duchenne muscular dystrophy (DMD) show alterations in the metabolism of fatty molecules, amino acids, and carnitine, a nutrient helping cells produce energy, relative to children without this disease, a study from China reports. Differences in the gut microbiome, the collection of microorganisms living in…

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, 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…