Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents.

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作者： Holly,Landrum Peay ^[1] ; Ryan,Fischer ^[2] ; Janice P,Tzeng ^[1] ; Sharon E,Hesterlee ^[3] ; Carl,Morris ^[4] ; Amy,Strong Martin ^[5] ; Colin,Rensch ^[2] ; Edward,Smith ^[6] ; Valeria,Ricotti ^[4] ; Katherine,Beaverson ^[7] ; Hannah,Wand ^[8] ; Carol,Mansfield ^[9]

作者单位： Center for Newborn Screening, Ethics, and Disability Studies, RTI International, Research Triangle Park, North Caroilina, United States of America. ^[1] Parent Project Muscular Dystrophy, Hackensack, New Jersey, United States of America. ^[2] Lion Therapeutics, Asklepios BioPharmaceutical, Inc., Research Triangle Park, North Carolina, United States of America. ^[3] Solid Biosciences, Cambridge, Massachusetts, United States of America. ^[4] Center for Duchenne Muscular Dystrophy at UCLA, University of California Los Angeles, Los Angeles, California, United States of America. ^[5] Duke University School of Medicine, Durham, North Carolina, United States of America. ^[6] Rare Disease Research Unit, Pfizer, Inc, Cambridge, Massachusetts, United States of America. ^[7] Stanford Healthcare and ClinGen, Sanford, California, United States of America. ^[8] RTI Health Solutions, RTI International, Research Triangle Park, North Carolina, United States of America. ^[9]