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Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER.
Thompson, William R; Manuel, Ryan; Abbruscato, Anthony; Carr, Jim; Campbell, John; Hornby, Brittany; Vaz, Frédéric M; Vernon, Hilary J.
Affiliation
  • Thompson WR; The Blalock-Taussig-Thomas Pediatric and Congenital Heart Center, Johns Hopkins University School of Medicine, Baltimore, MD.
  • Manuel R; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD.
  • Abbruscato A; Stealth BioTherapeutics, Inc., Newton, MA.
  • Carr J; Stealth BioTherapeutics, Inc., Newton, MA.
  • Campbell J; Stealth BioTherapeutics, Inc., Newton, MA.
  • Hornby B; Department of Physical Therapy, Kennedy Krieger, Baltimore, MD.
  • Vaz FM; Amsterdam UMC Location University of Amsterdam, Department of Clinical Chemistry and Pediatrics, Laboratory Genetic Metabolic Diseases, Emma Children's Hospital, Meibergdreef 9, Amsterdam, The Netherlands; Amsterdam Gastroenterology Endocrinology Metabolism, Inborn Errors of Metabolism, Amsterdam, T
  • Vernon HJ; Department of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD. Electronic address: hvernon1@jhmi.edu.
Genet Med ; 26(7): 101138, 2024 Jul.
Article in En | MEDLINE | ID: mdl-38602181
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligopeptides / Barth Syndrome Limits: Adolescent / Adult / Female / Humans / Male / Middle aged Language: En Journal: Genet Med Journal subject: GENETICA MEDICA Year: 2024 Document type: Article Affiliation country: Moldova Country of publication: United States
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Oligopeptides / Barth Syndrome Limits: Adolescent / Adult / Female / Humans / Male / Middle aged Language: En Journal: Genet Med Journal subject: GENETICA MEDICA Year: 2024 Document type: Article Affiliation country: Moldova Country of publication: United States