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Paper Details

Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment.
J Inherit Metab Dis
24
2021
Author NameAffiliation
Stephanie GrunewaldGreat Ormond Street Hospital, Institute of Child Health, NIHR Biomedical Research Center
Esther M MaierUniversity of Munich Children's Hospital
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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