Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
- Resource Type
- Article
- Authors
- Shieh, Perry B ; Kuntz, Nancy L; Dowling, James J; Müller-Felber, Wolfgang; Bönnemann, 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
- In The Lancet Neurology December 2023 22(12):1125-1139
- Subject
- Primary Research
Articles
- Language
- ISSN
- 1474-4422