Audentes Therapeutics, Inc., a biotechnology company committed to the development and commercialization of gene therapy products for patients with serious, rare diseases, today announced the presentation of results from ongoing preclinical studies of AT001 (AAV-MTM1), Audentes’ novel therapeutic in development for the treatment of X-linked Myotubular Myopathy (XLMTM). XLMTM is a rare, inherited disorder characterized by severe muscle weakness and respiratory impairment.
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