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Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
Smith, Edward C; Conklin, Laurie S; Hoffman, Eric P; Clemens, Paula R; Mah, Jean K; Finkel, Richard S; Guglieri, Michela; Tulinius, Mar; Nevo, Yoram; Ryan, Monique M; Webster, Richard; Castro, Diana; Kuntz, Nancy L; Kerchner, Laurie; Morgenroth, Lauren P; Arrieta, Adrienne; Shimony, Maya; Jaros, Mark; Shale, Phil; Gordish-Dressman, Heather; Hagerty, Laura; Dang, Utkarsh J; Damsker, Jesse M; Schwartz, Benjamin D; Mengle-Gaw, Laurel J; McDonald, Craig M.
Afiliación
  • Smith EC; Duke University, Durham, North Carolina, United States of America.
  • Conklin LS; ReveraGen Biopharma, Rockville, Maryland, United States of America.
  • Hoffman EP; Children's National Hospital, Washington, District of Columbia, United States of America.
  • Clemens PR; ReveraGen Biopharma, Rockville, Maryland, United States of America.
  • Mah JK; Binghamton University-SUNY, Binghamton, New York, United States of America.
  • Finkel RS; University of Pittsburgh and Department of Veterans Affairs Medical Center, Pittsburgh, Pennsylvania, United States of America.
  • Guglieri M; Alberta Children's Hospital, Calgary, Alberta, Canada.
  • Tulinius M; Nemours Children's Hospital, Orlando, Florida, United States of America.
  • Nevo Y; John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
  • Ryan MM; Queen Silvia Children's Hospital, Gothenburg, Sweden.
  • Webster R; Schneider Children's Medical Center, Tel Aviv University, Petah Tikvah, Israel.
  • Castro D; Royal Children's Hospital and Murdoch Children's Research Institute, Melbourne, Victoria, Australia.
  • Kuntz NL; The Children's Hospital at Westmead, Sydney, New South Wales, Australia.
  • Kerchner L; University of Texas Southwestern Medical Center, Dallas, Texas, United States of America.
  • Morgenroth LP; Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States of America.
  • Arrieta A; TRiNDS, Pittsburgh, Pennsylvania, United States of America.
  • Shimony M; TRiNDS, Pittsburgh, Pennsylvania, United States of America.
  • Jaros M; TRiNDS, Pittsburgh, Pennsylvania, United States of America.
  • Shale P; TRiNDS, Pittsburgh, Pennsylvania, United States of America.
  • Gordish-Dressman H; Summit Analytical, Denver, Colorado, United States of America.
  • Hagerty L; Summit Analytical, Denver, Colorado, United States of America.
  • Dang UJ; Children's National Hospital, Washington, District of Columbia, United States of America.
  • Damsker JM; ReveraGen Biopharma, Rockville, Maryland, United States of America.
  • Schwartz BD; Binghamton University-SUNY, Binghamton, New York, United States of America.
  • Mengle-Gaw LJ; ReveraGen Biopharma, Rockville, Maryland, United States of America.
  • McDonald CM; Camden Group, St. Louis, Missouri, United States of America.
PLoS Med ; 17(9): e1003222, 2020 09.
Article en En | MEDLINE | ID: mdl-32956407
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Pregnadienodioles / Distrofia Muscular de Duchenne / Actividad Motora Tipo de estudio: Clinical_trials Límite: Child / Child, preschool / Humans / Male Idioma: En Revista: PLoS Med Asunto de la revista: MEDICINA Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Pregnadienodioles / Distrofia Muscular de Duchenne / Actividad Motora Tipo de estudio: Clinical_trials Límite: Child / Child, preschool / Humans / Male Idioma: En Revista: PLoS Med Asunto de la revista: MEDICINA Año: 2020 Tipo del documento: Article País de afiliación: Estados Unidos