MTM1 and X-linked myotubular myopathy: Despite the safety of this approach, a recent phase I/II clinical trial (NCT03199469) for X-linked myotubular myopathy (MTM) utilizing AAV8 dose escalation of MTM1 protein (AT132) observed detrimental hepatic outcomes in humans when high viral loads (1 × 1014 vg/kg up to 3 × 1014 vg/kg) were used.