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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial.
Schoser, Benedikt; Roberts, Mark; Byrne, Barry J; Sitaraman, Sheela; Jiang, Hai; Laforêt, Pascal; Toscano, Antonio; Castelli, Jeff; Díaz-Manera, Jordi; Goldman, Mitchell; van der Ploeg, Ans T; Bratkovic, Drago; Kuchipudi, Srilakshmi; Mozaffar, Tahseen; Kishnani, Priya S.
Affiliation
  • Schoser B; Friedrich-Baur-Institut, Neurologische Klinik, Ludwig-Maximilians-Universität München, Munich, Germany. Electronic address: benedikt.schoser@med.uni-muenchen.de.
  • Roberts M; Salford Royal NHS Foundation Trust, Salford, UK.
  • Byrne BJ; University of Florida, Gainesville, FL, USA.
  • Sitaraman S; Amicus Therapeutics, Philadelphia, PA, USA.
  • Jiang H; Amicus Therapeutics, Philadelphia, PA, USA.
  • Laforêt P; Raymond-Poincaré Hospital, Garches, France.
  • Toscano A; University of Messina, Messina, Italy.
  • Castelli J; Amicus Therapeutics, Philadelphia, PA, USA.
  • Díaz-Manera J; Unitat de Malalties Neuromusculars Servei de Neurologia, Hospital de la Santa Creu i Sant Pau de Barcelona, Barcelona, Spain.
  • Goldman M; Amicus Therapeutics, Philadelphia, PA, USA.
  • van der Ploeg AT; ErasmusMC University Medical Center, Rotterdam, Netherlands.
  • Bratkovic D; PARC Research Clinic, Royal Adelaide Hospital, Adelaide, SA, Australia.
  • Kuchipudi S; Amicus Therapeutics, Philadelphia, PA, USA.
  • Mozaffar T; University of California, Irvine, CA, USA.
  • Kishnani PS; Duke University Medical Center, Durham, NC, USA.
Lancet Neurol ; 20(12): 1027-1037, 2021 12.
Article in En | MEDLINE | ID: mdl-34800400
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II Type of study: Clinical_trials / Prognostic_studies Limits: Adolescent / Humans Language: En Journal: Lancet Neurol Journal subject: NEUROLOGIA Year: 2021 Document type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycogen Storage Disease Type II Type of study: Clinical_trials / Prognostic_studies Limits: Adolescent / Humans Language: En Journal: Lancet Neurol Journal subject: NEUROLOGIA Year: 2021 Document type: Article