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INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy

James J. Dowling,Wolfgang Mueller-Felber,Barbara K. Smith,Carsten G. Bonnemann,Nancy L. Kuntz,Francesco Muntoni,Laurent Servais,Lindsay N. Alfano,Alan H. Beggs,Deborah A. Bilder,Astrid Blaschek,Tina Duong,Robert J. Graham,Minal Jain,Michael W. Lawlor,Jun Lee,Julie Coats,Charlotte Lilien,Linda P. Lowes,Victoria MacBean,Sarah Neuhaus,Mojtaba Noursalehi,Teresa Pitts, Caroline Finlay,Sarah Christensen,Gerrard Rafferty,Andreea M. Seferian,Etsuko Tsuchiya,Emma S. James,Weston Miller,Bryan Sepulveda,Maria Candida Vila,Suyash Prasad,Salvador Rico,Perry B. Shieh

Journal of neuromuscular diseases（2022）

Cited 6|Views44
Key words
X-linked myotubular myopathy,centronuclear myopathy,neuromuscular diseases,respiratory failure,ventilators,mechanical,motor disorders
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