News

The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell therapy designed to treat heart disease in Duchenne muscular dystrophy (DMD), by the end of August. The FDA agreed this year to review an application to approve deramiocel for…

People with myotonic dystrophy (DM) use more healthcare resources and face higher associated costs than patients without the disease, according to a U.S. study that analyzed a large database of insurance claims. After diagnosis, DM patients are more likely to be hospitalized and visit an emergency department, researchers found.

The Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency known as CHMP for short, has recommended the approval of Duvyzat (givinostat) as a treatment for people with Duchenne muscular dystrophy (DMD), ages 6 and older, who are able to walk. The European…

CureDuchenne will host its annual Futures National Conference May 22-25 in San Antonio, as part of an effort to provide education and offer connections to members of the Duchenne and Becker muscular dystrophy communities. Under the theme “Together We Thrive,” the event will share information on research and…

It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics, was approved by the U.S. Food and Drug Administration (FDA), marking the first time a gene therapy for DMD was authorized in the U.S. Since its…

The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies…

The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to delpacibart etedesiran, known as del-desiran, an investigational therapy from Avidity Biosciences now in clinical testing for myotonic dystrophy type 1 (DM1). This designation is intended to support the development of therapies for rare…

Health Canada has agreed to review an application by Kye Pharmaceuticals seeking the approval of vamorolone for treating Duchenne muscular dystrophy (DMD) — and granted it priority review. Priority review shortens the review period from the standard 10 months to six. It’s given to therapies that have the…

An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with Duchenne muscular dystrophy (DMD) in ongoing clinical trials, according to developer Sarepta Therapeutics. The determination comes after recruitment and dosing in certain clinical trials were paused after a young man who…

KER-065, a potential treatment for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases, showed a good safety profile in a Phase 1 clinical trial. No serious side effects were seen in the trial, which tested single and multiple doses of KER-065 in healthy volunteers, and no major safety issues…