Skip to Main Content

Paper Details

Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Lancet Neurol
9
2023
Author NameAffiliation
Francesco MuntoniGreat Ormond Street Hospital Biomedical Research Centre, University College London Institute of Child Health
Alan H BeggsThe Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School
  • 1 - 2

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