Fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy for pantothenate kinase-associated neurodegeneration: Mechanism of action and efficacy in nonclinical models

• Daniel Elbaum ,

  Roles Conceptualization, Methodology, Writing – original draft, Writing – review & editing

  * E-mail: Daniel.Elbaum@retrophin.com

  Affiliation Research and Development, Retrophin Inc., Cambridge, Massachusetts, United States of America

  http://orcid.org/0000-0002-6012-7965

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• Maria G. Beconi,

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

  Current address: Goldfinch Biopharma Inc., Cambridge, Massachusetts, United States of America

  Affiliation Research and Development, Retrophin Inc., Cambridge, Massachusetts, United States of America

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• Edith Monteagudo,

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

  Affiliation Preclinical Research, IRBM Science Park SpA, Pomezia, Rome, Italy

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• Annalise Di Marco,

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

  Affiliation In-vitro Pharmacology, IRBM Science Park SpA, Pomezia, Rome, Italy

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• Maria S. Quinton,

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

  Current address: Rodin Therapeutics, Cambridge, Massachusetts, United States of America

  Affiliation Research and Development, Retrophin Inc., Cambridge, Massachusetts, United States of America

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• Kathryn A. Lyons,

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

  Affiliation Independent consultant, Holland, New York, United States of America

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• Andrew Vaino,

  Roles Conceptualization, Methodology, Writing – original draft, Writing – review & editing

  Current address: LB Pharmaceuticals, New York, New York, United States of America

  Affiliation Research and Development, Retrophin Inc., Cambridge, Massachusetts, United States of America

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• Steven Harper

  Roles Conceptualization, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Medicinal Chemistry, IRBM Science Park SpA, Pomezia, Rome, Italy

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