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Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study.
Feldt-Rasmussen, Ulla; Hughes, Derralynn; Sunder-Plassmann, Gere; Shankar, Suma; Nedd, Khan; Olivotto, Iacopo; Ortiz, Damara; Ohashi, Toya; Hamazaki, Takashi; Skuban, Nina; Yu, Julie; Barth, Jay A; Nicholls, Kathleen.
Afiliação
  • Feldt-Rasmussen U; Department of Medical Endocrinology and Metabolism, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark. Electronic address: ufeldt@rh.dk.
  • Hughes D; Department of Haematology, Royal Free London NHS Foundation Trust and University College London, London, UK.
  • Sunder-Plassmann G; Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna, Vienna, Austria.
  • Shankar S; Department of Pediatrics, University of California-Davis, Davis, CA, USA.
  • Nedd K; Infusion Associates, Grand Rapids, MI, USA.
  • Olivotto I; Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy.
  • Ortiz D; Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, PA, USA.
  • Ohashi T; Jikei University Hospital, Tokyo, Japan.
  • Hamazaki T; Department of Pediatrics, Osaka City University Graduate School of Medicine, Osaka, Japan.
  • Skuban N; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
  • Yu J; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
  • Barth JA; Amicus Therapeutics, Inc., Cranbury, NJ, USA.
  • Nicholls K; Department of Nephrology, Royal Melbourne Hospital, University of Melbourne, Parkville, VIC, Australia.
Mol Genet Metab ; 131(1-2): 219-228, 2020.
Article em En | MEDLINE | ID: mdl-33012654
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Fabry / 1-Desoxinojirimicina / Terapia de Reposição de Enzimas / Rim Idioma: En Ano de publicação: 2020 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Doença de Fabry / 1-Desoxinojirimicina / Terapia de Reposição de Enzimas / Rim Idioma: En Ano de publicação: 2020 Tipo de documento: Article