Clinical trial evaluated gene therapy resamirigene bilparvovec in children with X-linked myotubular myopathy, indicating its potential benefits.
What is this summary about? This summary describes the results of a research study (clinical trial) called ASPIRO that was published in the Lancet Neurology in 2023. This study looked at an investigational gene therapy called resamirigene bilparvovec (also known as AT132) as a possible treatment for children with a disease called X-linked myotubular myopathy (abbreviated as XLMTM).
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Shieh et al. (2025) studied this question.
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