Tag: <span>Duchenne</span>

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Teenage girl gets long-awaited Duchenne treatment

Sarah Jenssen is the rarest of the rare — a girl with Duchenne muscular dystrophy, which almost always afflicts boys, as STAT wrote about in 2019. She wasn’t eligible to enroll in clinical trials for cutting-edge Duchenne therapies, but her family was elated when the FDA this year approved a gene therapy called Elevidys from Sarepta...

December 8, 2024December 9, 2024by In News
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Scientists uncover a novel approach to treating Duchenne muscular dystrophy

SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE Scientists at Sanford Burnham Prebys Medical Discovery Institute, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore in Rome have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring (fibrosis). The study,...