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Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study.

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Title: Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study.
Authors: Carrillo N; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA., Malicdan MC; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Leoyklang P; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Shrader JA; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA., Joe G; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA., Slota C; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA., Perreault J; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA., Heiss JD; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA., Class B; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA., Liu CY; Radiology and Imaging Sciences, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA., Bradley K; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Jodarski C; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Ciccone C; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Driscoll C; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Parks R; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA., Van Wart S; Enhanced Pharmacodynamics LLC, Buffalo, NY, USA., Bayman L; Clinical Trials Statistical and Data Management Center, University of Iowa, Iowa City, IA, USA., Coffey CS; Clinical Trials Statistical and Data Management Center, University of Iowa, Iowa City, IA, USA., Quintana M; Berry Consultants LLC, Austin, TX, USA., Berry SM; Berry Consultants LLC, Austin, TX, USA., Huizing M; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Gahl WA; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA. gahlw@mail.nih.gov.
Source: Genetics in medicine : official journal of the American College of Medical Genetics [Genet Med] 2021 Nov; Vol. 23 (11), pp. 2067-2075. Date of Electronic Publication: 2021 Jul 13.
Publication Type: Clinical Trial, Phase II; Journal Article; Research Support, N.I.H., Extramural; Research Support, N.I.H., Intramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 9815831 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1530-0366 (Electronic) Linking ISSN: 10983600 NLM ISO Abbreviation: Genet Med Subsets: MEDLINE
Database: MEDLINE Ultimate
FullText Text:
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PubType: Academic Journal
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  Data: Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study.
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  Data: <searchLink fieldCode="AU" term="%22Carrillo+N%22">Carrillo N</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Malicdan+MC%22">Malicdan MC</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Leoyklang+P%22">Leoyklang P</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Shrader+JA%22">Shrader JA</searchLink>; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Joe+G%22">Joe G</searchLink>; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Slota+C%22">Slota C</searchLink>; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Perreault+J%22">Perreault J</searchLink>; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Heiss+JD%22">Heiss JD</searchLink>; National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Class+B%22">Class B</searchLink>; Therapeutics for Rare and Neglected Diseases, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Liu+CY%22">Liu CY</searchLink>; Radiology and Imaging Sciences, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Bradley+K%22">Bradley K</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Jodarski+C%22">Jodarski C</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Ciccone+C%22">Ciccone C</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Driscoll+C%22">Driscoll C</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Parks+R%22">Parks R</searchLink>; Department of Rehabilitation Medicine, NIH Clinical Center, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Van+Wart+S%22">Van Wart S</searchLink>; Enhanced Pharmacodynamics LLC, Buffalo, NY, USA.<br /><searchLink fieldCode="AU" term="%22Bayman+L%22">Bayman L</searchLink>; Clinical Trials Statistical and Data Management Center, University of Iowa, Iowa City, IA, USA.<br /><searchLink fieldCode="AU" term="%22Coffey+CS%22">Coffey CS</searchLink>; Clinical Trials Statistical and Data Management Center, University of Iowa, Iowa City, IA, USA.<br /><searchLink fieldCode="AU" term="%22Quintana+M%22">Quintana M</searchLink>; Berry Consultants LLC, Austin, TX, USA.<br /><searchLink fieldCode="AU" term="%22Berry+SM%22">Berry SM</searchLink>; Berry Consultants LLC, Austin, TX, USA.<br /><searchLink fieldCode="AU" term="%22Huizing+M%22">Huizing M</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Gahl+WA%22">Gahl WA</searchLink>; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA. gahlw@mail.nih.gov.
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  Data: <searchLink fieldCode="JN" term="%229815831%22">Genetics in medicine : official journal of the American College of Medical Genetics</searchLink> [Genet Med] 2021 Nov; Vol. 23 (11), pp. 2067-2075. <i>Date of Electronic Publication: </i>2021 Jul 13.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Elsevier%22">Elsevier </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>9815831 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1530-0366 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2210983600%22">10983600 </searchLink><i>NLM ISO Abbreviation: </i>Genet Med <i>Subsets: </i>MEDLINE
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