U.S., July 3 -- ClinicalTrials.gov registry received information related to the study (NCT07680751) titled 'European Cystinosis Cohort 2' on June 22.
Brief Summary: This European observational cohort follows patients with cystinosis, a rare lysosomal storage disease caused by CTNS mutations leading to cystine accumulation and multisystem involvement. It aims to describe the long-term clinical course under current treatments, focusing on renal and extra-renal complications, survival, and quality of life. It also evaluates treatment effects and explores biomarkers, including inflammatory markers, with biobanking for future research.
Study Start Date: July 01
Study Type: OBSERVATIONAL
Condition:
Cystinosis
Recruitment Status: NOT_YET_REC...