학술논문
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
Document Type
Journal
Author
Shieh, Perry B.; Kuntz, Nancy L.; Dowling, James J.; Mueller-Felber, Wolfgang; Bonnemann, Carsten G.; Seferian, Andreea M.; Servais, Laurent; Smith, Barbara K.; Muntoni, Francesco; Blaschek, Astrid; Foley, A. Reghan; Saade, Dimah N.; Neuhaus, Sarah; Alfano, Lindsay N.; Beggs, Alan H.; Buj-Bello, Ana; Childers, Martin K.; Duong, Tina; Graham, Robert J.; Jain, Minal; Coats, Julie; Macbean, Vicky; James, Emma S.; Lee, Jun; Mavilio, Fulvio; Miller, Weston; Varfaj, Fatbardha; Murtagh, Michael; Han, Cong; Noursalehi, Mojtaba; Lawlor, Michael W.; Prasad, Suyash; Rico, Salvador
Source
Subject
Language
English
ISSN
14744465