Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study

What is this summary about? This summary describes the results of a research study (clinical trial) called ASPIRO that was published in the <i>Lancet Neurology</i> in 2023. This study looked at an investigational gene therapy called <b>resamirigene bilparvovec</b> (also known as AT132) as a possible treatment for children with a disease called X-linked <b>myotubular myopathy</b> (abbreviated as XLMTM).