To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued ...
May 14, 2025
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The U.S. Food and Drug Administration (FDA) has chosen Dr. Vinay Prasad, a professor at the University of California-San Francisco, to lead its Center for Biologics Evaluation and Research.
May 7, 2025
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A new study led by researchers at the Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD), a severe condition that ...
Apr 30, 2025
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Many diseases are caused by a missing or defective copy of a single gene. For decades, scientists have been working on gene therapy treatments that could cure such diseases by delivering a new copy of the missing genes to ...
Apr 28, 2025
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A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a large-scale clinical ...
Apr 8, 2025
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New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing severe muscle degeneration, also profoundly affects the brain, leading to cognitive and behavioral ...
Mar 28, 2025
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For millions of people, losing muscle isn't just about weakness; it's about losing independence. Whether caused by Duchenne muscular dystrophy, aging or other degenerative conditions, muscle loss can make everyday activities—like ...
Mar 26, 2025
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A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment.
Mar 19, 2025
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Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%.
Mar 18, 2025
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Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The disease is chronic and begins in childhood. The life expectancy of affected patients is significantly ...
Feb 12, 2025
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