XLMTM

AcronymDefinition
XLMTMX-Linked Myotubular Myopathy
References in periodicals archive ?
This analysis confirmed and expanded upon the understanding of the significant disease burden of XLMTM on patients, families and the healthcare system.
Summary: Administration of AT132 produced at 500L scale demonstrated an encouraging safety profile and high potency, providing support for its advancement into clinical testing for XLMTM patients.
This analysis confirmed and expanded upon the understanding of the medical burden of XLMTM on patients, families and the healthcare system.
XLMTM is caused by mutations in the MTM1 gene, which encodes a protein called myotubularin.