News

The U.S. Food and Drug Administration (FDA) has given orphan drug designation to JUV-161, Juvena Therapeutics’ lead treatment candidate for myotonic dystrophy type 1 (DM1). Orphan drug status is designed to encourage the development of therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides…

Sarepta Therapeutics has started screening participants for a Phase 3 clinical trial called EMERGENE that will test its gene therapy candidate SRP-9003 in children with limb-girdle muscular dystrophy type 2E (LGMD2E). The open-label trial, also known as SRP-9003-301, aims to recruit 15 patients, age 4 and older, with or without…

SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to therapies that are designed to improve medical care for rare disorders, specifically defined…

Agamree (vamorolone) has been approved in the U.K. for treating Duchenne muscular dystrophy (DMD) in patients 4 and older. This approval by the Medicines and Healthcare products Regulatory Agency (MHRA) follows a similar decision in the European Union. In the U.S., Agamree is approved for patients…

Race to End Duchenne, the signature fundraising program from Parent Project Muscular Dystrophy (PPMD), has presented Team Michael James with the 2024 “Racing for Every Future Team” award, while Michael Napoli was this year’s “Make Every Day Count” award recipient. Race to End Duchenne supports PPMD’s mission to cure…

Dosing has begun in a Phase 2 clinical trial evaluating PepGen‘s PGN-EDO51 — a treatment candidate for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping. The trial, called CONNECT1-EDO51 (NCT06079736), will evaluate the safety and preliminary efficacy of the experimental therapy in about 10 patients,…

New trial data announced by Dyne Therapeutics show two experimental treatments for muscular dystrophy from the company’s FORCE platform appear to be working as intended in early clinical studies. Both DYNE-251 for Duchenne muscular dystrophy (DMD), being tested in a trial dubbed DELIVER, and DYNE-101 for…

Throughout 2023, Muscular Dystrophy News Today worked diligently to cover new scientific research, treatment developments, and clinical studies for muscular dystrophy (MD). Here are the 10 most-read stories of that year. No. 10 – With EMBARK trial data, Sarepta seeks to expand Elevidys’ approval Elevidys (delandistrogene moxeparvovec-roki) is a…

People with Becker muscular dystrophy (BMD) caused by specific mutations tend to be younger when they first start using a wheelchair, a new study reports. The study, “Natural history of Becker muscular dystrophy: a multicenter study of 225 patients,” also provides new insights into the frequency of…

Disease progression with Becker muscular dystrophy (BMD) varies considerably, and depends on a patient’s age — with progression largely seen only in adults — and type of mutation, a three-year study of BMD’s natural history shows. “We … provide evidence that decline/improvement in function would be hard to measure…