Rare diseases often go undiagnosed or untreated in parts of Africa. A project seeks to change that
Ndeye Lam visits the cemetery often, praying and gently touching the seashells laid out across her daughter's gravesite.
Apr 27, 2025
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Musculoskeletal Diseases: News and Research on Muscular Dystrophy, Duchenne
MeSH tree: C05.651.534.500.300
Setback in gene therapy for Duchenne muscular dystrophy as immune system emerges as key barrier
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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Duchenne muscular dystrophy's impact on the brain may be reversible
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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New protein target identified for muscle repair and regeneration
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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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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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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