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Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety, Efficacy and Pathology with AT132

Michael Lawlor,Benedikt Schoser,Caroline Sewry,Marta Margeta,James Dowling,Carsten Bonnemann,Nancy Kuntz,Wolfgang Muller-Felber,Mo Noursalehi, Salvador Rico,Laurent Servais,Perry Shieh,Barbara Smith,Suyash Prasad

JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY（2019）

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