Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

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Bibliographic Details
Title: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.
Authors: Byrne BJ; Department of Pediatrics in the College of Medicine, University of Florida, Gainesville, FL, United States., Parenti G; Metabolic Unit, Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy.; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy., Schoser B; Friedrich-Baur-Institute, Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany., van der Ploeg AT; Erasmus MC University Medical Center, Rotterdam, Netherlands., Do H; M6P-Therapeutics, St Louis, MO, United States., Fox B; Amicus Therapeutics, Inc., Princeton, NJ, United States., Goldman M; Amicus Therapeutics, Inc., Princeton, NJ, United States., Johnson FK; Amicus Therapeutics, Inc., Princeton, NJ, United States., Kang J; Metrum Research Group, Tariffville, CT, United States., Mehta N; Amicus Therapeutics, Inc., Princeton, NJ, United States., Mondick J; Incyte Corporation, Wilmington, DE, United States., Sheikh MO; Amicus Therapeutics, Inc., Princeton, NJ, United States., Sitaraman Das S; Amicus Therapeutics, Inc., Princeton, NJ, United States., Tuske S; Amicus Therapeutics, Inc., Princeton, NJ, United States., Brudvig J; Amicus Therapeutics, Inc., Princeton, NJ, United States., Weimer JM; Amicus Therapeutics, Inc., Princeton, NJ, United States., Mozaffar T; Department of Neurology, University of California, Irvine, Irvine, CA, United States.
Source: Frontiers in neurology [Front Neurol] 2024 Oct 18; Vol. 15, pp. 1451512. Date of Electronic Publication: 2024 Oct 18 (Print Publication: 2024).
Publication Type: Journal Article
Journal Info: Publisher: Frontiers Research Foundation Country of Publication: Switzerland NLM ID: 101546899 Publication Model: eCollection Cited Medium: Print ISSN: 1664-2295 (Print) Linking ISSN: 16642295 NLM ISO Abbreviation: Front Neurol Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1664-2295
DOI:10.3389/fneur.2024.1451512