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

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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
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  Data: <searchLink fieldCode="AU" term="%22Byrne+BJ%22">Byrne BJ</searchLink>; Department of Pediatrics in the College of Medicine, University of Florida, Gainesville, FL, United States.<br /><searchLink fieldCode="AU" term="%22Parenti+G%22">Parenti G</searchLink>; Metabolic Unit, Department of Translational Medical Sciences, University of Naples Federico II, Naples, Italy.; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy.<br /><searchLink fieldCode="AU" term="%22Schoser+B%22">Schoser B</searchLink>; Friedrich-Baur-Institute, Department of Neurology, LMU University Hospital, LMU Munich, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22van+der+Ploeg+AT%22">van der Ploeg AT</searchLink>; Erasmus MC University Medical Center, Rotterdam, Netherlands.<br /><searchLink fieldCode="AU" term="%22Do+H%22">Do H</searchLink>; M6P-Therapeutics, St Louis, MO, United States.<br /><searchLink fieldCode="AU" term="%22Fox+B%22">Fox B</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Goldman+M%22">Goldman M</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Johnson+FK%22">Johnson FK</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Kang+J%22">Kang J</searchLink>; Metrum Research Group, Tariffville, CT, United States.<br /><searchLink fieldCode="AU" term="%22Mehta+N%22">Mehta N</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Mondick+J%22">Mondick J</searchLink>; Incyte Corporation, Wilmington, DE, United States.<br /><searchLink fieldCode="AU" term="%22Sheikh+MO%22">Sheikh MO</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Sitaraman+Das+S%22">Sitaraman Das S</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Tuske+S%22">Tuske S</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Brudvig+J%22">Brudvig J</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Weimer+JM%22">Weimer JM</searchLink>; Amicus Therapeutics, Inc., Princeton, NJ, United States.<br /><searchLink fieldCode="AU" term="%22Mozaffar+T%22">Mozaffar T</searchLink>; Department of Neurology, University of California, Irvine, Irvine, CA, United States.
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  Data: <searchLink fieldCode="JN" term="%22101546899%22">Frontiers in neurology</searchLink> [Front Neurol] 2024 Oct 18; Vol. 15, pp. 1451512. <i>Date of Electronic Publication: </i>2024 Oct 18 (<i>Print Publication: </i>2024).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Frontiers+Research+Foundation%22">Frontiers Research Foundation </searchLink><i>Country of Publication: </i>Switzerland <i>NLM ID: </i>101546899 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>1664-2295 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2216642295%22">16642295 </searchLink><i>NLM ISO Abbreviation: </i>Front Neurol <i>Subsets: </i>PubMed not MEDLINE
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