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This is an observational study examining a multi-omic approach to identify new biomarkers in individuals with early-stage Charcot-Marie-Tooth 1A disease (CMT1A). People aged 10-30 with a genetic diagnosis of CMT1A (or a clinical diagnosis with a genetic diagnosis in affected relatives) who can walk with or without support can take part. It is a study with no assigned phase and is currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion criteria: * collaborative children, adolescents and young adults aged 10-30 years * genetic diagnosis of CMT1A, or clinical diagnosis and genetic diagnosis in affected relatives * able to walk with/ without support. Exclusion Criteria: * neuromuscular disorders other than CMT1A * concomitant disease preventing correct patient evaluation and contraindication to qMRI