Clinical Trial: A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational




Official Title: The RECENSUS Study: A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

Brief Summary: This retrospective medical chart review (RECENSUS) of approximately 100 XLMTM patients (with a goal to obtain 50 deceased and 20 living records) will provide further knowledge about the clinical manifestations and recorded medical management of XLMTM and potentially inform the design of future therapeutic intervention studies.