Natural History Study for Patients With Nemaline Myopathy in Belgium

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.

NCT Number	NCT07201636
Lead Sponsor	Centre Hospitalier Universitaire de Liege Collaborators: Centre Hospitalier Régional de la Citadelle, SYSNAV
Conditions	Nemaline Myopathy
Enrollment	10 participants
Start Date	2026-08
Primary Completion	2029-04 (estimated)
Study Completion	2029-08 (estimated)
Updated on ClinicalTrials.gov	2026-03-25