Sarepta's gene therapy under scrutiny after patient death
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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Nervous System Diseases: News and Research on Muscular Dystrophy, Duchenne
MeSH tree: C10.668.491.175.500.300
Patient dies following muscular dystrophy gene therapy, Sarepta reports
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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Genetic mechanisms underlying Duchenne muscular dystrophy point to potential treatments
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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Personalized therapy for rare genetic diseases: Patient-derived organoids offer new hope
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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New insights into protein roles in Duchenne muscular dystrophy
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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Gene editing tool reduces Alzheimer's plaque precursor in mice
A new gene editing tool that helps cellular machinery skip parts of genes responsible for diseases has been applied to reduce the formation of amyloid-beta plaque precursors in a mouse model of Alzheimer's disease, researchers ...
Dec 23, 2024
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New drug shows potential in treating Duchenne muscular dystrophy
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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Researchers discover zip code that allows proteins to hitch a ride around the body
Researchers at The Ottawa Hospital and the University of Ottawa have discovered an 18-digit code that allows proteins to attach themselves to exosomes—tiny pinched-off pieces of cells that travel around the body and deliver ...
Dec 11, 2024
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Evaluating long-term survival and cardiac efficacy of a gene therapy for Duchenne muscular dystrophy
A recent study published in the journal Human Gene Therapy evaluated the long-term survival and cardiac efficacy of the gene therapy delandistrogene moxeparvovec in a rat model of Duchenne muscular dystrophy (DMD).
Dec 4, 2024
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$3.2 million per dose Elevidys fails to meet primary endpoint in phase 3 trial
A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings published in Nature Medicine. A News ...
