Participant has a diagnosis of XLMTM resulting from a genetically confirmed mutation in the MTM1 gene based on genetic test reports.
Participant requires some mechanical ventilatory support (e.g., ranging from 24 hours per day full-time mechanical ventilation, to non-invasive support such as continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) during sleeping hours)
Participant (as applicable) and/or parent(s)/carer is willing to comply with the recommended schedule of assessments.
You may not be if
Participant is currently enrolled in an interventional study designed to treat XLMTM.
Clareo Health | A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition