A new treatment platform developed by researchers at the University of Texas MD Anderson Cancer Center was able to deliver messenger RNA (mRNA) of the full-length DMD gene into preclinical models of Duchenne muscular dystrophy, ...
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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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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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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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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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Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the use of personalized antisense oligonucleotides (ASOs). This innovative approach has shown promising ...
Jan 24, 2025
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A new study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for understanding and treating Duchenne Muscular ...
Jan 2, 2025
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A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration.
Dec 16, 2024
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Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The genes that are dysfunctional in muscular dystrophies are often extremely ...
Nov 14, 2024
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