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http://bura.brunel.ac.uk/handle/2438/31987| Title: | Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study |
| Authors: | Shieh, P Hughes, W Wood, M Beggs, A Lawlor, M Coats, J Varfaj, F Graham, R Kuntz, N Dowling, J Muller-Felber, W Bonnemann, C Buj Bello, A Servais, L MacBean, V Muntoni, F Foley, AR Blaschek, A James, E Seferian, A Alfano, L Duong, T Noursalehi, M Miller, W Lee, J Prasad, S Rico, S |
| Keywords: | gene therapy;X-linked myotubular myopathy;resamirigene bilparvovec |
| Issue Date: | 18-Sep-2025 |
| Publisher: | SAGE Publications |
| Citation: | Shieh, P. et al. (2025) 'Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study', Therapeutic Advances in Rare Disease, 6, pp. 1 - 12. doi: 10.1177/26330040251362885. |
| 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). |
| Description: | Availability of data and materials: Details for how researchers may request access to anonymized participant level data, trial-level data, and protocols from Astellas sponsored clinical trials can be found at https://www.clinicaltrials.astellas.com/transparency/. |
| URI: | https://bura.brunel.ac.uk/handle/2438/31987 |
| DOI: | https://doi.org/10.1177/26330040251362885 |
| Other Identifiers: | ORCiD: Perry B. Shieh https://orcid.org/0000-0001-7145-7663 ORCiD: Marie Wood https://orcid.org/0009-0000-1665-3004 ORCiD: Michael W. Lawlor https://orcid.org/0000-0003-0731-3950 ORCiD: Victoria MacBean https://orcid.org/0000-0003-0268-2693 |
| Appears in Collections: | Dept of Health Sciences Research Papers |
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|---|---|---|---|---|
| FullText.pdf | Copyright © The Author(s), 2025. Rights and permissions: Creative Commons License (CC BY 4.0). This article is distributed under the terms of the Creative Commons Attribution 4.0 License (https://creativecommons.org/licenses/by/4.0/) which permits any use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage). | 1.24 MB | Adobe PDF | View/Open |
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