News

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…

The U.S. Food and Drug Administration (FDA) has approved vamorolone for treating people with Duchenne muscular dystrophy (DMD), ages 2 and older. The therapy will be marketed as Agamree. “We strongly believe that this novel steroid has the transformational potential to make a significant difference for patients living with…

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…

After three years of virtual academies, applications are now open for Duchenne Patient Academy 2023, an in-person training event for patient advocates for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). The academy, presented by the World Duchenne Organization (WDO) and the Duchenne Data…

The U.S. Food and Drug Administration (FDA) has lifted its clinical hold on PGN-EDODM1, a medicine being developed by PepGen for myotonic dystrophy type 1, or DM1 — a subtype of the most common form of muscular dystrophy. With the hold lifted, PepGen is ready to…

A European Medicines Agency (EMA) committee has recommended that vamorolone be approved in the European Union to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older. The recommendation, from the EMA’s Committee for Medicinal Products for Human Use (CHMP), now will be reviewed by the European…

People with myotonic dystrophy type 1 (DM1) reported gains in measures of muscle strength after being treated with AOC 1001 in a clinical trial. That’s according to new data announced by Avidity Biosciences, the developer of AOC 1001. The findings “reinforce our belief in the potential of AOC 1001…