Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial-Reference-Cited by-同舟云学术

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial

Published:2023-12 Issue:12 Volume:22 Page:1125-1139
ISSN:1474-4422
Container-title:The Lancet Neurology
language:en
Short-container-title:The Lancet Neurology

Author:

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

Funder

Astellas Pharma Inc

Publisher

Elsevier BV

Subject

Neurology (clinical)

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3. A multicenter, retrospective medical record review of X-linked myotubular myopathy: the RECENSUS study;Beggs;Muscle Nerve,2018

4. X-linked myotubular myopathy;Lawlor;Neuromuscul Disord,2021

5. Genotype-phenotype correlations in X-linked myotubular myopathy;McEntagart;Neuromuscul Disord,2002

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