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X-linked Myotubular Myopathy clinical trials at UC Health
2 in progress, 1 open to new patients

  • Gene Transfer Clinical Study in X-Linked Myotubular Myopathy

    open to eligible males ages up to 5 years

    This is a Phase 1/2, multinational, open-label, ascending-dose, delayed-treatment concurrent control clinical study to evaluate the safety and preliminary efficacy of AT132 in subjects with X-Linked Myotubular Myopathy aged less than 5 years old. Subjects will receive a single dose of AT132 and will be followed for safety and efficacy for 5 years

    at UCLA

  • A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects

    Sorry, in progress, not accepting new patients

    This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger. Many of these clinically relevant measures have not yet been routinely assessed in this population and may provide important insight on the natural history of XLMTM and for future evaluation of potential therapies.

    at UCLA

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