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

Saved in:
Bibliographic Details
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
Description
ISSN:1530-0366
DOI:10.1038/s41436-021-01259-x