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...
Tag: <span>Duchenne</span>
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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,...
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Human ‘chimeric’ cells restore crucial protein in Duchenne muscular Dystrophy
Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy — a rare but fatal form of muscular dystrophy — were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease. The findings are reported by researchers...