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 Lancet Neurology in 2023. This study looked at an investigational gene therapy called resamirigene bilparvovec (also known as AT132) as a possible treatment for...

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Auteurs principaux:	Perry B. Shieh, Wendy Hughes, Marie Wood, Alan H. Beggs, Michael W. Lawlor, Julie Coats, Fatbardha Varfaj, Robert J. Graham, Nancy L. Kuntz, James J. Dowling, Wolfgang Müller-Felber, Carsten G. Bönnemann, Ana Buj Bello, Laurent Servais, Vicky MacBean, Francesco Muntoni, A. Reghan Foley, Astrid Blaschek, Emma S. James, Andreea Seferian, Lindsay N. Alfano, Tina Duong, Mojtaba Noursalehi, Weston Miller, Jun Lee, Suyash Prasad, Salvador Rico
Format:	Artigo
Langue:	Inglês
Publié:	SAGE Publishing 2025-09-01
Collection:	Therapeutic Advances in Rare Disease
Accès en ligne:	https://doi.org/10.1177/26330040251362885
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