Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Authors
Kristl G. Claeys (ORCID: https://orcid.org/0000-0001-9937-443X)
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Kristl G. Claeys, Priya Sunil Kishnani, Benedikt G. H. Schoser, Mazen M. Dimachkie et al.
Citations
8 citations
Journal
Journal of Patient-Reported Outcomes
Topics
Lysosomal Storage Disorders Research
Field-Weighted Citation Impact (FWCI)
2.61
article
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Kristl G. Claeys, Priya Sunil Kishnani, Benedikt G. H. Schoser, Mazen M. Dimachkie, Hani Kushlaf, Simon Shohet, Barry J. Byrne, Jordi Díaz‐Manera, António Toscano, Tahseen Mozaffar, Mark Roberts, Fred Holdbrook, Agnieszka Kopiec, Noemi Hummel
article en
8 citations
Abstract
No abstract available for this paper.
Publication Tag
Journal of Patient-Reported OutcomesVol. 8(1)
Institutions
University of Messina (IT), Amicus Therapeutics (United Kingdom) (GB), 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), Duke Medical Center (US), University of Kansas Medical Center (US), Muscular Dystrophy UK (GB), Friedrich Baur Stiftung (DE), Amicus Therapeutics (United States) (US), Instituto de Investigación de Enfermedades Raras (ES), Ludwig-Maximilians-Universität München (DE), Newcastle University (GB), University of Cincinnati Medical Center (US), KU Leuven (BE)