Skip to Main Content

Paper Details

Safety and efficacy of N-acetylmannosamine (ManNAc) in patients with GNE myopathy: an open-label phase 2 study.
Genet Med
19
2021
GNE, GNE myopathy, ManNAc, Myopathy, N-acetylmannosamine, N-acetylneuraminic acid, Neu5Ac, genetic muscle disease, patients
Author NameAffiliation
May Christine V MalicdanNational Human Genome Research Institute, National Institutes of Health
May Christine V MalicdanNational Human Genome Research Institute, National Institutes of Health
Christopher S CoffeyClinical Trials Statistical and Data Management Center, University of Iowa
Scott M BerryBerry Consultants LLC
William A GahlNational Human Genome Research Institute, National Institutes of Health
William A GahlNational Human Genome Research Institute, National Institutes of Health
  • 1 - 6

Datasets

ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link
ClinicalTrials.govClinicalTrials.gov, a registry and results database of of privately and publicly funded clinical studies conducted around the world, is operated by the U.S. National Library of Medicine (NLM). Structured study information is provided, voluntarily or to meet legal and policy requirements, by the sponsor or investigator responsible for the research. NLM does not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.Link