Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial
Resource Type
Article
Authors
Lawlor, Michael W. ; Schoser, Benedikt ; Margeta, Marta ; Sewry, Caroline A. ; Jones, Karra A. ; Shieh, Perry B. ; Kuntz, Nancy L. ; Smith, Barbara K. ; Dowling, James J. ; Müller-Felber, Wolfgang ; Bönnemann, Carsten G. ; Seferian, Andreea M. ; Blaschek, Astrid ; Neuhaus, Sarah ; Foley, A. Reghan ; Saade, Dimah N. ; Tsuchiya, Etsuko ; Qasim, Ummulwara R. ; Beatka, Margaret ; Prom, Mariah J. ; Ott, Emily ; Danielson, Susan ; Krakau, Paul ; Kumar, Suresh N. ; Meng, Hui ; Vanden Avond, Mark ; Wells, Clive ; Gordish-Dressman, Heather ; Beggs, Alan H. ; Christensen, Sarah ; Conner, Edward ; James, Emma S. ; Lee, Jun ; Sadhu, Chanchal ; Miller, Weston ; Sepulveda, Bryan ; Varfaj, Fatbardha ; Prasad, Suyash ; Rico, Salvador
Source
In eBioMedicine January 2024 99
Subject
Language
ISSN
2352-3964