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Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association, provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics.

Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy.

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association, provided an update on its efforts to employ Artificial Intelligence in revolutionizing the development and bioproduction of gene therapies.

Genethon today announced The Lancet Neurology published online preliminary results of a clinical trial conducted by Astellas Pharma using a gene therapy developed by Genethon for X-linked myotubular myopathy, a rare genetic muscle disease affecting 1 in 50,000 newborn boys.

Genethon, a pioneer and leader in gene therapy research and development for rare genetic diseases, announced positive Phase 1/2 clinical trial results of its gene therapy, GNT-0003, for Crigler-Najjar syndrome were published in The New England Journal of Medicine.

Genethon is a leader in developing gene therapies for rare and ultra-rare diseases

In a European Phase I/II clinical trial, the experimental drug for treating Crigler-Najjar syndrome reduced bilirubin below toxic threshold in the liver with a single intravenous injection.