Treatment Trials

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A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)
Description

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.