Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of <scp>PROPEL</scp>
Authors
Mark Jonathan Roberts (ORCID: https://orcid.org/0000-0001-7513-1281)
Kristl G. Claeys (ORCID: https://orcid.org/0000-0001-9937-443X)
Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL
Mark Jonathan Roberts, Kristl G. Claeys, Priya Sunil Kishnani, Benedikt G. H. Schoser et al.
Citations
4 citations
Journal
Muscle & Nerve
Topics
Lysosomal Storage Disorders Research
Field-Weighted Citation Impact (FWCI)
4.66
article
Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL
Mark Jonathan Roberts, Kristl G. Claeys, Priya Sunil Kishnani, Benedikt G. H. Schoser, Mazen M. Dimachkie, Sheela Sitaraman Das, Hani Kushlaf, Barry J. Byrne, Jordi Díaz‐Manera, Drago Bratkovic, António Toscano, Paula R. Clemens, Tahseen Mozaffar, Mitchell H. Goldman, Jeff Castelli, Fred Holdbrook, Pascal Laforêt
article en
4 citations
Abstract
No abstract available for this paper.
Publication Tag
Muscle & NerveVol. 72(2)
Institutions
University of Messina (IT), Royal Adelaide Hospital (AU), University of Pittsburgh (US), University of California, Irvine (US), Salford Royal NHS Foundation Trust (GB), Hospital de Sant Pau (ES), University of Florida (US), Centre for Biomedical Network Research on Rare Diseases (ES), Centro de Investigación Biomédica en Red (ES), Duke Medical Center (US), University of Kansas Medical Center (US), Friedrich Baur Stiftung (DE), VA Pittsburgh Healthcare System (US), Amicus Therapeutics (United States) (US), Hôpital Raymond-Poincaré (FR), University of Cincinnati (US), Ludwig-Maximilians-Universität München (DE), Newcastle University (GB), KU Leuven (BE)