Status: Recruiting
Natural History Study for Patients With Nemaline Myopathy in Spain
The objective of this natural history study is to comprehensively characterize the disease progression and clinical features of nemaline myopathies. The study aims to establish a well-defined cohort of patients in Spain, enabling long-term follow-up and facilitating recruitment for future clinical trials.
Participants needed: 100
Trial details
Biological sex: AllType: ObservationalSponsor: Hospital Universitari Vall d'Hebron Research InstituteUpdated: Mar 23, 2026Locations: 1Duration: 5 Years
Eligibility criteria
Patients with a confirmed clinical and genetic diagnosis of MN (mutations in ACT... [+1]