Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
Titel:
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
Auteur:
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
Verschenen in:
The lancet neurology
Paginering:
Jaargang 22 () nr. 12 pagina's 1125-1139
Jaar:
2023
Inhoud:
Uitgever:
The Author(s). Published by Elsevier Ltd. This is an Open Access article under the CC BY-NC-ND 4.0 license