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A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study.

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Title: A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study.
Authors: Beggs AH; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, 300 Longwood Avenue Boston, Massachusetts, USA., Byrne BJ; Children's Research Institute, University of Florida, Gainesville, Gainesville, Florida, USA., De Chastonay S; Cure CMD, Torrance, California, USA., Haselkorn T; EpiMetrix, Inc., Los Altos, California, USA., Hughes I; Royal Manchester Children's Hospital, Manchester, United Kingdom., James ES; Audentes Therapeutics, San Francisco, California, USA., Kuntz NL; Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA., Simon J; Statistics Collaborative, Washington, DC., Swanson LC; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, 300 Longwood Avenue Boston, Massachusetts, USA., Yang ML; Children's Hospital Colorado, Aurora, Colorado, USA., Yu ZF; Statistics Collaborative, Washington, DC., Yum SW; Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA., Prasad S; Audentes Therapeutics, San Francisco, California, USA.
Source: Muscle & nerve [Muscle Nerve] 2018 Apr; Vol. 57 (4), pp. 550-560. Date of Electronic Publication: 2017 Dec 22.
Publication Type: Journal Article; Multicenter Study; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: John Wiley & Sons Country of Publication: United States NLM ID: 7803146 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1097-4598 (Electronic) Linking ISSN: 0148639X NLM ISO Abbreviation: Muscle Nerve Subsets: MEDLINE
Database: MEDLINE Ultimate
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Description
ISSN:1097-4598
DOI:10.1002/mus.26018