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Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02).
Byrne, Barry J; Schoser, Benedikt; Kishnani, Priya S; Bratkovic, Drago; Clemens, Paula R; Goker-Alpan, Ozlem; Ming, Xue; Roberts, Mark; Vorgerd, Matthias; Sivakumar, Kumaraswamy; van der Ploeg, Ans T; Goldman, Mitchell; Wright, Jacquelyn; Holdbrook, Fred; Jain, Vipul; Benjamin, Elfrida R; Johnson, Franklin; Das, Sheela Sitaraman; Wasfi, Yasmine; Mozaffar, Tahseen.
Afiliação
  • Byrne BJ; University of Florida, Gainesville, FL, USA. barry.byrne@ufl.edu.
  • Schoser B; Friedrich-Baur-Institute at the Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany.
  • Kishnani PS; Duke University Medical Center, Durham, NC, USA.
  • Bratkovic D; PARC Research Clinic, Royal Adelaide Hospital, Adelaide, SA, Australia.
  • Clemens PR; Department of Neurology, University of Pittsburgh School of Medicine and VA Pittsburgh Healthcare System, Pittsburgh, PA, USA.
  • Goker-Alpan O; Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, USA.
  • Ming X; Neurology, Rutgers New Jersey Medical School, Newark, NJ, USA.
  • Roberts M; Guam Regional Medical City, Dededo, Guam.
  • Vorgerd M; Salford Royal NHS Foundation Trust, Salford, UK.
  • Sivakumar K; Department of Neurology, University Hospital Bergmannsheil, Heimer Institute for Muscle Research, Bochum, Germany.
  • van der Ploeg AT; Neuromuscular Clinic and Research Center, Phoenix, AZ, USA.
  • Goldman M; Erasmus MC University Medical Center, Rotterdam, Netherlands.
  • Wright J; Amicus Therapeutics, Inc., Princeton, NJ, USA.
  • Holdbrook F; Amicus Therapeutics, Inc., Princeton, NJ, USA.
  • Jain V; Amicus Therapeutics, Inc., Princeton, NJ, USA.
  • Benjamin ER; Amicus Therapeutics, Inc., Princeton, NJ, USA.
  • Johnson F; Amicus Therapeutics, Inc., Princeton, NJ, USA.
  • Das SS; Amicus Therapeutics, Inc., Princeton, NJ, USA.
  • Wasfi Y; Amicus Therapeutics, Inc., Princeton, NJ, USA.
  • Mozaffar T; Amicus Therapeutics, Inc., Princeton, NJ, USA.
J Neurol ; 271(4): 1787-1801, 2024 Apr.
Article em En | MEDLINE | ID: mdl-38057636
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Propionatos / Doença de Depósito de Glicogênio Tipo II / 1-Desoxinojirimicina Limite: Adult / Humans Idioma: En Revista: J Neurol Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Estados Unidos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Propionatos / Doença de Depósito de Glicogênio Tipo II / 1-Desoxinojirimicina Limite: Adult / Humans Idioma: En Revista: J Neurol Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Estados Unidos