INCEPTUS multinational, prospective, natural history, run-in study of males with X-linked myotubular myopathy

Perry Shieh,Wolfgang Mueller-Felber,Weston Miller, Bryan Sepulveda,James Dowling

Genetics in Medicine（2022）

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摘要

XLMTM is an ultra-rare, life-threatening congenital myopathy, caused by variants in the MTM1 gene, and characterized by profound muscle weakness at birth; with no approved treatments. We present results from INCEPTUS (NCT02704273), a prospective, non-interventional XLMTM study.

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x-linked

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