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Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Alexion Pharmaceuticals, Inc., Ara Parseghian Medical Research Foundation, BioMarin Pharmaceutical, Cure Sanfilippo Foundation, Danas Angels Research Trust (DART), Mirum Pharmaceuticals, Inc., Orchard Therapeutics, Passage Bio, Inc., Genzyme, a Sanofi Company, Sio Gene Therapies, Takeda Pharmaceuticals North America, Inc., The FireFly Fund, The Noah's Hope - Hope 4 Bridget Family Foundations, Travere Therapeutics, Inc., and Ultragenyx Pharmaceutical Inc

Joanne Kurtzberg, MD, Professor of Pediatrics

Pulse Infoframe Ltd.

Sanofi and Chung-Hsing Wang, Attending Physicians

Deepa Soundara Rajan, Associate Professor

University of North Carolina, Chapel Hill, The John Merck Fund, Duke University, Wake Forest University, North Carolina Department of Health and Human Services, National Center for Advancing Translational Sciences (NCATS), Cure SMA, The National Fragile X Foundation, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Asuragen, Inc., Sarepta Therapeutics, Inc., Muscular Dystrophy Association, The Leona M. and Harry B. Helmsley Charitable Trust, Juvenile Diabetes Research Foundation, Janssen Pharmaceuticals, GeneDx, and Illumina, Inc.

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Eline C. B. Eskes, MD

Duke University

Sanofi

Engage Health Inc. and Icahn School of Medicine at Mount Sinai

Huiwen Zhang, MD, Associate Prof.

Washington University School of Medicine and Weill Medical College of Cornell University