U.S., Oct. 2 -- ClinicalTrials.gov registry received information related to the study (NCT07201636) titled 'Natural History Study for Patients With Nemaline Myopathy in Belgium' on Aug. 21.
Brief Summary: This is a prospective, longitudinal, observational study designed to characterize the natural history of Nemaline Myopathy (NM), a rare congenital neuromuscular disorder. The study will follow up to 10 participants of any age with genetically confirmed NM over a period of three years. Data will be collected during routine annual hospital visits and include clinical, motor, respiratory, and quality-of-life assessments. The goal is to improve clinical trial readiness by identifying disease-specific outcome measures and potential biomarkers....