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Moving Forward after Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

Moving Forward after Two Deaths in a Gene Therapy Trial of Myotubular Myopathy 1 The sponsor of this clinical study, Audentes Therapeutics (which was acquired by Japanโ€™s Astellas Pharma in 2019), had generated impressive efficacy studies in a canine model of MTM 2 and conducted non-primate (NHP) safety studies before advancing into the clinic. 3 The initial cohort of six pediatric MTM patients dosed at 1ร—10 14 genome copies (GC)/kg also showed encouraging results. However, all three patien...
United States Astellas Pharma Jesse Gelsinger American Society For Gene Dose Intravenous Administration Disease Community

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