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INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy
James J. Dowling*, Wolfgang Müller-Felber, Barbara K. Smith, Carsten G. Bönnemann, Nancy L. Kuntz, Francesco Muntoni, Laurent Servais, Lindsay N. Alfano, Alan H. Beggs, Deborah A. Bilder, Astrid Blaschek, Tina Duong, Robert J. Graham, Minal Jain, Michael W. Lawlor, Jun Lee, Julie Coats, Charlotte Lilien, Linda P. Lowes, Victoria MacBean
*Corresponding author for this work
- The Hospital for Sick Children Research Institute
- Ludwig Maximilian University of Munich
- University of Florida
- Epilepsy Research Branch, National Institute on Neurological Disorders and Stroke
- Children's Memorial Hospital
- UCL Great Ormond Street Institute of Child Health
- Hôpital Armand Trousseau
- CHU of Liege
- Nationwide Children’s Hospital
- Harvard Medical School
- University of Utah
- Stanford University
- NIH National Institutes of Health
- Medical College of Wisconsin
- Formerly of Astellas Gene Therapies (Formerly Audentes Therapeutics)
- Astellas Gene Therapies
- Brunel University London
- University of Louisville
- King's College London
- University of California at Los Angeles
Research output: Contribution to journal › Article › peer-review
12
Citations
(Scopus)