The U.S. Food and Drug Administration (FDA) has cleared Precision Biosciences to launch a clinical trial testing its gene-editing therapy candidate PBGENE-DMD in people with Duchenne muscular dystrophy (DMD) caused by certain mutations. Precision announced it had received a “Study May Proceed notification” from the U.S. regulatory agency…
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A Phase 3 trial testing SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD), will start dosing before the end of March, according to Solid Biosciences. The trial follows a successful meeting with the U.S. Food and Drug Administration (FDA), during which the company and the agency aligned…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to Dyscorban (ifetroban), Cumberland Pharmaceuticals’ treatment candidate for heart problems in Duchenne muscular dystrophy (DMD). Dyscorban has been tested in the Phase 2 FIGHT DMD trial (NCT03340675), with data showing that it improved heart…
A research collaboration led by the Muscular Dystrophy Association (MDA) aims to create a molecular map of human muscle regeneration, a project that could accelerate the development of muscle repair-based therapies for people with muscular dystrophy (MD). Abigail Mackey, PhD, a professor of muscle physiology and regeneration at…
Three years after receiving the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) in a clinical trial, boys with Duchenne muscular dystrophy (DMD) are showing sustained improvements in physical function compared with what would be expected without treatment, according to new data announced by Sarepta Therapeutics, Elevidys’ developer. “ELEVIDYS is…
For people with Duchenne muscular dystrophy (DMD), 2026 promises to be a year of tremendous excitement, with several new therapies poised for likely approval in the U.S., according to DMD advocates. “It’s an exciting time,” Debra Miller, founder CEO of CureDuchenne, said in an interview with Muscular Dystrophy…
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…
Recent Posts
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- New Phase 3 trial of Duchenne gene therapy SGT-003 to begin dosing
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