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Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases.
Koenig, Mary Kay; Russo, Sam Nick; McBride, Kim L; Bjornsson, Hans Tomas; Gunnarsdottir, Brynja Bjork; Goldstein, Amy; Falk, Scott A.
Afiliação
  • Koenig MK; The University of Texas McGovern Medical School, Center for the Treatment of Pediatric Neurodegenerative Disease Houston Texas USA.
  • Russo SN; The University of Texas McGovern Medical School, Center for the Treatment of Pediatric Neurodegenerative Disease Houston Texas USA.
  • McBride KL; Division of Genetic and Genomic Medicine and the Heart Center Nationwide Children's Hospital, Department of Pediatrics College of Medicine Ohio State University Columbus Ohio USA.
  • Bjornsson HT; Landspitali University Hospital Reykjavik Iceland.
  • Gunnarsdottir BB; Faculty of Medicine University of Iceland Reykjavik Iceland.
  • Goldstein A; Mckusick-Nathans Department of Genetic Medicine Johns Hopkins University Baltimore Maryland USA.
  • Falk SA; Landspitali University Hospital Reykjavik Iceland.
JIMD Rep ; 64(1): 65-70, 2023 Jan.
Article em En | MEDLINE | ID: mdl-36636586
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: JIMD Rep Ano de publicação: 2023 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: JIMD Rep Ano de publicação: 2023 Tipo de documento: Article