Improved Muscle Strength and Attainment of Motor Milestones in Boys with X-linked Myotubular Myopathy ( XLMTM) Administered Single-dose Gene Replacement Therapy (resamirigene Bilparvovec)

Lindsay N. Alfano,James J. Dowling,Perry Shieh, Wolfgang Mueller-Felber, Carsten G. Boennemann, Andreea M. Seferian,Barbara K. Smith, Jun Lee, Mojtaba Noursalehi,Salvador Rico, Suyash Prasad, Julie Coats, Weston Miller,Tina Duong,Nancy L. Kuntz

PEDIATRICS(2022)

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