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

Abstract

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).

Keywords

gene therapy

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Footnotes

Acknowledgements

The authors thank the patients and families with XLMTM who participated in the INCEPTUS and ASPIRO studies, and are grateful for support from the XLMTM patient advocacy community.

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

Abstract

Keywords

Footnotes

Acknowledgements

Declarations