News

Using a “pothole-filling” strategy that combines the precision of DNA-like strands with the versatility of small molecules, scientists have designed a new type of therapy that can target the mutant genetic material that causes myotonic dystrophy type 1 (DM1). This hybrid approach allows scientists to stabilize tangled RNA structures…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to (Z)-endoxifen for Duchenne muscular dystrophy (DMD), providing Atossa Therapeutics with incentives to encourage further research into its DMD treatment candidate. The company said it plans to continue working closely with the FDA as it develops…

In boys with Duchenne muscular dystrophy (DMD), executive skills — essentially the brain’s self-management tools, such as self-control, emotional regulation, and working memory — appear largely typical at age 5, begin to lag around age 8, and show signs of partial catch-up by early adolescence. Those are the findings…

The Muscular Dystrophy Association (MDA) has opened applications for its 2026 college scholarship program, which provides financial support for students living with muscular dystrophy or a related neuromuscular disease. With the program now in its third year, the scholarships are available to students who are pursuing or planning…

The Muscular Dystrophy Association (MDA) announced that registration is now open for its 2026 summer camp program. The program is open to kids and teens ages 8 to 17 who have muscular dystrophy or a related neuromuscular disease. Families can submit applications for their campers via an…

Throughout 2025, Muscular Dystrophy News Today brought you daily coverage of the latest muscular dystrophy (MD)-related clinical research and scientific breakthroughs. Here are the year’s top 10 most-read stories we published last year, each with a brief description. No. 10 — FSHD patients sought for first EPI-321 clinical…

Duchenne muscular dystrophy (DMD) causes dysfunction of muscle cells that help move blood through the circulatory system, a study found. The changes associated with DMD included dysregulated activity of some genes and altered dynamics and structure of mitochondria, the cell’s powerhouses. “These findings highlight the importance of targeting vascular…

Every newborn in the United States should now be screened for Duchenne muscular dystrophy (DMD), according to a recommendation from the U.S. Department of Health and Human Services (HHS). This federal decision marks a turning point for rare disease advocacy, potentially ending the “diagnostic odyssey” that many families face when…

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to (Z)-endoxifen for Duchenne muscular dystrophy (DMD), a step that could allow its developer, Atossa Therapeutics, to qualify for a future priority review benefit for another experimental treatment in its pipeline. “This designation is an important regulatory…

Dyne Therapeutics said it plans to ask the U.S. Food and Drug Administration (FDA) to approve DYNE-251, its exon-skipping therapy for people with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, after a Phase 1/2 trial showed encouraging results. Data from the registrational expansion cohort of the…