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INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
Dowling, James J; Müller-Felber, Wolfgang; Smith, Barbara K; Bönnemann, Carsten G; Kuntz, Nancy L; Muntoni, Francesco; Servais, Laurent; Alfano, Lindsay N; Beggs, Alan H; Bilder, Deborah A; Blaschek, Astrid; Duong, Tina; Graham, Robert J; Jain, Minal; Lawlor, Michael W; Lee, Jun; Coats, Julie; Lilien, Charlotte; Lowes, Linda P; MacBean, Victoria; Neuhaus, Sarah; Noursalehi, Mojtaba; Pitts, Teresa; Finlay, Caroline; Christensen, Sarah; Rafferty, Gerrard; Seferian, Andreea M; Tsuchiya, Etsuko; James, Emma S; Miller, Weston; Sepulveda, Bryan; Vila, Maria Candida; Prasad, Suyash; Rico, Salvador; Shieh, Perry B.
Afiliación
  • Dowling JJ; Hospital for Sick Children, Toronto, Canada.
  • Müller-Felber W; Dr. v. Haunersches Kinderspital, Klinikum der Universität München, Munich, Germany.
  • Smith BK; University of Florida, Gainesville, FL, USA.
  • Bönnemann CG; National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD, USA.
  • Kuntz NL; Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.
  • Muntoni F; National Institute for Health Research (NIHR) Great Ormond Street (GOS) Hospital Biomedical Research Centre, University College London Institute of Child Health, London, UK.
  • Servais L; I-Motion, Hôpital Armand Trousseau, Paris, France.
  • Alfano LN; Division of Child Neurology, Reference Center for Neuromuscular Diseases, Department of Pediatrics, University Hospital Liège & University of Liège, Belgium.
  • Beggs AH; Nationwide Children's Hospital, Columbus, OH, USA.
  • Bilder DA; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Blaschek A; University of Utah, Salt Lake City, UT, USA.
  • Duong T; Dr. v. Haunersches Kinderspital, Klinikum der Universität München, Munich, Germany.
  • Graham RJ; Stanford University, Palo Alto, CA, USA.
  • Jain M; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Lawlor MW; NIH Hatfield Clinical Research Center, Bethesda, MD, USA.
  • Lee J; Medical College of Wisconsin, Milwaukee, WI, USA.
  • Coats J; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.
  • Lilien C; Astellas Gene Therapies (formerly Audentes Therapeutics), San Francisco, CA, USA.
  • Lowes LP; I-Motion, Hôpital Armand Trousseau, Paris, France.
  • MacBean V; Nationwide Children's Hospital, Columbus, OH, USA.
  • Neuhaus S; Brunel University London, London, UK and King's College 32 London, London, UK.
  • Noursalehi M; National Institute of Neurological Disorders and Stroke, NIH, Bethesda, MD, USA.
  • Pitts T; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.
  • Finlay C; University of Louisville, Louisville, KY, USA.
  • Christensen S; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.
  • Rafferty G; University of Louisville, Louisville, KY, USA.
  • Seferian AM; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.
  • Tsuchiya E; University of Louisville, Louisville, KY, USA.
  • James ES; King's College London, London, UK.
  • Miller W; I-Motion, Hôpital Armand Trousseau, Paris, France.
  • Sepulveda B; Hospital for Sick Children, Toronto, Canada.
  • Vila MC; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.
  • Prasad S; University of Louisville, Louisville, KY, USA.
  • Rico S; Astellas Gene Therapies (formerly Audentes Therapeutics), San Francisco, CA, USA.
  • Shieh PB; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics) San Francisco, CA, USA.
J Neuromuscul Dis ; 9(4): 503-516, 2022.
Article en En | MEDLINE | ID: mdl-35694931
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Calidad de Vida / Miopatías Estructurales Congénitas Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies Límite: Child, preschool / Humans / Male Idioma: En Revista: J Neuromuscul Dis Año: 2022 Tipo del documento: Article País de afiliación: Canadá
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Calidad de Vida / Miopatías Estructurales Congénitas Tipo de estudio: Clinical_trials / Observational_studies / Prognostic_studies Límite: Child, preschool / Humans / Male Idioma: En Revista: J Neuromuscul Dis Año: 2022 Tipo del documento: Article País de afiliación: Canadá