A gene therapy alliance in X-linked chronic granulomatous disease with Orchard Therapeutics
Orchard Therapeutics (“Orchard”), a clinical-stage biotechnology company dedicated to transforming the lives of patients with rare disorders through innovative gene therapies announces today a strategic alliance with Généthon, to develop gene therapy for X-linked chronic granulomatous disease (“X-CGD”)….
(read more)Audentes Therapeutics announces dosing of first patient in ASPIRO for the treatment of X-Linked Myotubular Myopathy
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(read more)Restore muscle strength in Duchenne muscular dystrophy through microdystrophin
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(read more)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…
(read more)New step towards the treatment of myotubular myopathy: gene therapy restores strength and prolongs lives in affected dogs
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(read more)ASGCT 18th Annual Meeting 2015: Ana Buj Bello receives the Outstanding New Investigator Award
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(read more)Selecta and Genethon Collaborate to Create Next Generation Gene Therapies Using Selecta’s Synthetic Vaccine Particle Platform
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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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