Miglustat: A First-In-Class Enzyme Stabilizer for Late-Onset Pompe Disease (P10-2.012)
To demonstrate the benefits of the two-component therapy cipaglucosidase alfa (cipa) plus oral miglustat (mig) in patients with late-onset Pompe disease (LOPD).
Authors
- Giancarlo Parenti (ORCID: https://orcid.org/0000-0002-6287-5748)
- Robert J. Hopkin (ORCID: https://orcid.org/0000-0003-0770-5516)
- Jon J. Brudvig (ORCID: https://orcid.org/0000-0002-3324-6125)
- Vipul Jain (ORCID: https://orcid.org/0009-0001-2757-2135)
- Priya Sunil Kishnani (ORCID: https://orcid.org/0000-0001-8251-909X)
- Benedikt G. H. Schoser (ORCID: https://orcid.org/0000-0002-2757-8131)
- Mazen M. Dimachkie (ORCID: https://orcid.org/0000-0002-7148-989X)
- Barry J. Byrne
- Tahseen Mozaffar (ORCID: https://orcid.org/0000-0002-1230-0188)
- Jufen Zhang (ORCID: https://orcid.org/0000-0003-2704-6212)
- Frederick Holdbrook
- Mark Roberts
- Brian W. Fox
- Franklin K. Johnson (ORCID: https://orcid.org/0000-0002-5426-8171)
- Ans van der Ploeg
Institutions
- Cincinnati Children's Hospital Medical Center (US)
- Amicus Therapeutics (United Kingdom) (GB)
- University of California, Irvine (US)
- Salford Royal NHS Foundation Trust (GB)
- Erasmus MC (NL)
- University of Florida (US)
- Duke Medical Center (US)
- University of Kansas Medical Center (US)
- Friedrich Baur Stiftung (DE)
- University of Naples Federico II (IT)
- Ludwig-Maximilians-Universität München (DE)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000211414
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00