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

• Holly Landrum Peay ,

  Roles Conceptualization, Data curation, Formal analysis, Funding acquisition, Investigation, Methodology, Project administration, Supervision, Writing – original draft, Writing – review & editing

  * E-mail: Hpeay@rti.org

  Affiliation Center for Newborn Screening, Ethics, and Disability Studies, RTI International, Research Triangle Park, North Caroilina, United States of America

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• Ryan Fischer,

  Roles Conceptualization, Funding acquisition, Investigation, Methodology, Project administration, Writing – original draft, Writing – review & editing

  Affiliation Parent Project Muscular Dystrophy, Hackensack, New Jersey, United States of America

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• Janice P. Tzeng,

  Roles Conceptualization, Formal analysis, Investigation, Methodology, Project administration, Writing – original draft, Writing – review & editing

  Affiliation Center for Newborn Screening, Ethics, and Disability Studies, RTI International, Research Triangle Park, North Caroilina, United States of America

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• Sharon E. Hesterlee ,

  Contributed equally to this work with: Sharon E. Hesterlee, Carl Morris, Amy Strong Martin, Colin Rensch, Edward Smith, Valeria Ricotti, Katherine Beaverson

  Roles Conceptualization, Methodology, Writing – review & editing

  Affiliation Lion Therapeutics, Asklepios BioPharmaceutical, Inc., Research Triangle Park, North Carolina, United States of America

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• Carl Morris ,

  Contributed equally to this work with: Sharon E. Hesterlee, Carl Morris, Amy Strong Martin, Colin Rensch, Edward Smith, Valeria Ricotti, Katherine Beaverson

  Roles Conceptualization, Methodology, Writing – review & editing

  Affiliation Solid Biosciences, Cambridge, Massachusetts, United States of America

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• Amy Strong Martin ,

  Contributed equally to this work with: Sharon E. Hesterlee, Carl Morris, Amy Strong Martin, Colin Rensch, Edward Smith, Valeria Ricotti, Katherine Beaverson

  Roles Conceptualization, Methodology, Writing – review & editing

  Affiliation Center for Duchenne Muscular Dystrophy at UCLA, University of California Los Angeles, Los Angeles, California, United States of America

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• Colin Rensch ,

  Contributed equally to this work with: Sharon E. Hesterlee, Carl Morris, Amy Strong Martin, Colin Rensch, Edward Smith, Valeria Ricotti, Katherine Beaverson

  Roles Conceptualization, Methodology, Writing – review & editing

  Affiliation Parent Project Muscular Dystrophy, Hackensack, New Jersey, United States of America

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• Edward Smith ,

  Contributed equally to this work with: Sharon E. Hesterlee, Carl Morris, Amy Strong Martin, Colin Rensch, Edward Smith, Valeria Ricotti, Katherine Beaverson

  Roles Conceptualization, Methodology, Writing – review & editing

  Affiliation Duke University School of Medicine, Durham, North Carolina, United States of America

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• Valeria Ricotti ,

  Contributed equally to this work with: Sharon E. Hesterlee, Carl Morris, Amy Strong Martin, Colin Rensch, Edward Smith, Valeria Ricotti, Katherine Beaverson

  Roles Conceptualization, Methodology, Writing – review & editing

  Affiliation Solid Biosciences, Cambridge, Massachusetts, United States of America

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• Katherine Beaverson ,

  Contributed equally to this work with: Sharon E. Hesterlee, Carl Morris, Amy Strong Martin, Colin Rensch, Edward Smith, Valeria Ricotti, Katherine Beaverson

  Roles Conceptualization, Methodology, Writing – review & editing

  Affiliation Rare Disease Research Unit, Pfizer, Inc, Cambridge, Massachusetts, United States of America

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• Hannah Wand,

  Roles Methodology, Writing – review & editing

  Affiliation Stanford Healthcare and ClinGen, Sanford, California, United States of America

  http://orcid.org/0000-0001-8662-3353

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• Carol Mansfield

  Roles Conceptualization, Investigation, Methodology, Supervision, Writing – review & editing

  Affiliation RTI Health Solutions, RTI International, Research Triangle Park, North Carolina, United States of America

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