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Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis.
Graham, Robert J; Muntoni, Francesco; Hughes, Imelda; Yum, Sabrina W; Kuntz, Nancy L; Yang, Michele L; Byrne, Barry J; Prasad, Suyash; Alvarez, Rachel; Genetti, Casie A; Haselkorn, Tmirah; James, Emma S; LaRusso, Laurie B; Noursalehi, Mojtaba; Rico, Salvador; Beggs, Alan H.
Affiliation
  • Graham RJ; Department of Anesthesiology, Critical Care and Pain Medicine, Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA beggs@enders.tch.harvard.edu robert.graham@childrens.harvard.edu.
  • Muntoni F; The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health and NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.
  • Hughes I; Royal Manchester Children's Hospital, Manchester, UK.
  • Yum SW; Children's Hospital of Philadelphia, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
  • Kuntz NL; Ann and Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
  • Yang ML; Children's Hospital Colorado, Aurora, Colorado, USA.
  • Byrne BJ; Children's Research Institute, University of Florida, Gainesville, Florida, USA.
  • Prasad S; Audentes Therapeutics, San Francisco, California, USA.
  • Alvarez R; Cure CMD, Torrance, California, USA.
  • Genetti CA; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  • Haselkorn T; EpiMetrix, Los Altos, California, USA.
  • James ES; Audentes Therapeutics, San Francisco, California, USA.
  • LaRusso LB; Chestnut Medical Communications, Walpole, Massachusetts, USA.
  • Noursalehi M; Audentes Therapeutics, San Francisco, California, USA.
  • Rico S; Audentes Therapeutics, San Francisco, California, USA.
  • Beggs AH; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA beggs@enders.tch.harvard.edu robert.graham@childrens.harvard.edu.
Arch Dis Child ; 105(4): 332-338, 2020 04.
Article in En | MEDLINE | ID: mdl-31484632

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Respiration, Artificial / Myopathies, Structural, Congenital Type of study: Observational_studies / Prognostic_studies / Risk_factors_studies / Screening_studies Limits: Child / Child, preschool / Female / Humans / Infant / Male / Newborn Language: En Journal: Arch Dis Child Year: 2020 Document type: Article Country of publication: Reino Unido

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Respiration, Artificial / Myopathies, Structural, Congenital Type of study: Observational_studies / Prognostic_studies / Risk_factors_studies / Screening_studies Limits: Child / Child, preschool / Female / Humans / Infant / Male / Newborn Language: En Journal: Arch Dis Child Year: 2020 Document type: Article Country of publication: Reino Unido