U.S., July 4 -- ClinicalTrials.gov registry received information related to the study (NCT07049588) titled 'Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease' on June 13.
Brief Summary: This is a 2-year follow-up study of a cohort of 35 CMT1A patients and 20 healthy volunteers. The main objective is identifying prognostic markers for CMT1A using multi-omics analysis. The study is recruiting subjects between the ages of 10 and 30.
The most common inherited neuropathy is Charcot-Marie-Tooth disease type 1A (CMT1A), caused by a duplication of the gene expressing PMP22. CMT1A patients develop symptoms in early childhood with variable progression and there is no established therapy until now. Therapy must start in chil...