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Please use this identifier to cite or link to this item: http://bura.brunel.ac.uk/handle/2438/31987
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dc.contributor.authorShieh, P-
dc.contributor.authorHughes, W-
dc.contributor.authorWood, M-
dc.contributor.authorBeggs, A-
dc.contributor.authorLawlor, M-
dc.contributor.authorCoats, J-
dc.contributor.authorVarfaj, F-
dc.contributor.authorGraham, R-
dc.contributor.authorKuntz, N-
dc.contributor.authorDowling, J-
dc.contributor.authorMuller-Felber, W-
dc.contributor.authorBonnemann, C-
dc.contributor.authorBuj Bello, A-
dc.contributor.authorServais, L-
dc.contributor.authorMacBean, V-
dc.contributor.authorMuntoni, F-
dc.contributor.authorFoley, AR-
dc.contributor.authorBlaschek, A-
dc.contributor.authorJames, E-
dc.contributor.authorSeferian, A-
dc.contributor.authorAlfano, L-
dc.contributor.authorDuong, T-
dc.contributor.authorNoursalehi, M-
dc.contributor.authorMiller, W-
dc.contributor.authorLee, J-
dc.contributor.authorPrasad, S-
dc.contributor.authorRico, S-
dc.date.accessioned2025-09-13T14:47:59Z-
dc.date.available2025-09-13T14:47:59Z-
dc.date.issued2025-09-18-
dc.identifierORCiD: Perry B. Shieh https://orcid.org/0000-0001-7145-7663-
dc.identifierORCiD: Marie Wood https://orcid.org/0009-0000-1665-3004-
dc.identifierORCiD: Michael W. Lawlor https://orcid.org/0000-0003-0731-3950-
dc.identifierORCiD: Victoria MacBean https://orcid.org/0000-0003-0268-2693-
dc.identifier.citationShieh, 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.en_US
dc.identifier.urihttps://bura.brunel.ac.uk/handle/2438/31987-
dc.descriptionAvailability 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/.en_US
dc.description.abstractWhat 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).en_US
dc.description.sponsorshipThe ASPIRO trial was sponsored by Astellas Gene Therapies. The authors acknowledge Lucy Smithers, PhD, and Sinead Stewart of Alpha (a division of Prime, Knutsford, UK), for medical writing and editorial support. This was funded by Astellas Gene Therapies in accordance with Good Publication Practice guidelines.en_US
dc.format.extent1 - 12-
dc.format.mediumElectronic-
dc.language.isoen_USen_US
dc.publisherSAGE Publicationsen_US
dc.rightsCreative Commons Attribution 4.0 International-
dc.rights.urihttps://creativecommons.org/licenses/by/4.0/-
dc.subjectgene therapyen_US
dc.subjectX-linked myotubular myopathyen_US
dc.subjectresamirigene bilparvovecen_US
dc.titleGene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO studyen_US
dc.typeArticleen_US
dc.date.dateAccepted2025-07-11-
dc.identifier.doihttps://doi.org/10.1177/26330040251362885-
dc.relation.isPartOfTherapeutic Advances in Rare Disease-
pubs.publication-statusPublished online-
pubs.volume6-
dc.identifier.eissn2633-0040-
dc.rights.licensehttps://creativecommons.org/licenses/by/4.0/legalcode.en-
dcterms.dateAccepted2025-07-11-
dc.rights.holderThe Author(s)-
Appears in Collections:Dept of Health Sciences Research Papers

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