Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
Perry B. Shieh(Boston Children's Hospital), Salvador Rico(Vital Therapies (United States)), Martin K. Childers(University of Washington), Fulvio Mavilio(The San Raffaele Telethon Institute for Gene Therapy), A. Seferian(Sorbonne Université), Nancy L. Kuntz(Lurie Children's Hospital), Tina Duong(Stanford University), Robert J. Graham(Boston Children's Hospital), Michael W. Lawlor(Medical College of Wisconsin), Wolfgang Müller‐Felber(Ludwig-Maximilians-Universität München), Cong Han(Astellas Pharma (United States)), Michael Murtagh(Vital Therapies (United States)), Sarah Neuhaus(National Institutes of Health), Lindsay N. Alfano(Nationwide Children's Hospital), Astrid Blaschek(Ludwig-Maximilians-Universität München), Alan H. Beggs(Boston Children's Hospital), Barbara K. Smith(University of Florida), Carsten G. Bönnemann(National Institute of Neurological Disorders and Stroke), Laurent Servais(University of Oxford), Mojtaba Noursalehi(Vital Therapies (United States)), D. Saade(University of Iowa Hospitals and Clinics), Weston P. Miller(University of Minnesota Children's Hospital), A. Reghan Foley(National Institutes of Health), Ana Buj‐Bello(Inserm), Jun Lee(Chosun University), Minal S. Jain(National Institutes of Health Clinical Center), Francesco Muntoni(Great Ormond Street Hospital), Julie Coats(Vital Therapies (United States)), Emma S. James(Vital Therapies (United States)), Suyash Prasad(Vital Therapies (United States)), Fatbardha Varfaj(Vital Therapies (United States))
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