New partnership for the treatment of Duchenne muscular disease (DMD)
Sarepta Therapeutics, a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare neuromuscular diseases, and Genethon, have signed a gene therapy research collaboration to jointly develop treatments for Duchenne…
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(read more)Evidence of efficacy of gene therapy in rodents affected by a rare genetic liver disease, Crigler-Najjar syndrome
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(read more)New gene therapy success in a rare disease of the immune system: Wiskott-Aldrich syndrome
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(read more)Effectiveness of innovative gene therapy treatment demonstrated in canine model of Duchenne muscular dystrophy
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(read more)GENETHON and ESTEVE announce agreement to manufacture the gene therapy for the treatment of Sanfilippo Syndrome
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(read more)Audentes Therapeutics and Genethon Announce Agreement to Develop Treatment for Severe Genetic Disease X‐Linked Myotubular Myopathy
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(read more)Efficacy of gene therapy demonstrated in canine and murine models of myotubular myopathy
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(read more)Généthon, the French AFM-Telethon laboratory, becomes the first not-for-profit to obtain authorization from ANSM to be a pharmaceutical manufacturer
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