The investigational cell therapy deramiocel slowed upper limb functional decline in boys and young men with Duchenne muscular dystrophy, data from the phase III HOPE-3 trial showed. At 12 months, the ...
Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité—Universitätsmedizin Berlin, have developed a promising gene-editing ...
A few short days after announcing an FDA pivot on a separate asset, REGENXBIO is planning to test the agency’s apparent ...
Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...
The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...
The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...
Sarepta Therapeutics is seeking to convert the accelerated approval of its therapeutic exon-skippers for Duchenne muscular dystrophy to full despite the drugs’ failure to improve motor function in a ...
Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...
The MarketWatch News Department was not involved in the creation of this content. New York, Aug. 23, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and Parent Project Muscular ...
Most parents wouldn’t be thrilled with the idea of their kids getting hooked up to an IV bag filled with trillions of viruses. But for Melanie Hennick, whose son, Connor, has Duchenne muscular ...
When Chase Finazzo was just a few years old, his parents noticed Chase was pretty clumsy. But they didn't think it was anything serious. "He would fall a lot. Not like a lot a lot. But he had trouble ...
WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the ...
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