Brunel University Research Archive(BURA) preserves and enables easy and open access to all
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Research contained within BURA is open access, although some publications may be subject
to publisher imposed embargoes. All awarded PhD theses are also archived on BURA.
Please use this identifier to cite or link to this item:
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: | 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, 0 (accepted, in press) |
| Abstract: | ... |
| Description: | ... |
| URI: | https://bura.brunel.ac.uk/handle/2438/31987 |
| ISSN: | 2633-0040 |
| Other Identifiers: | ORCiD: Victoria MacBean https://orcid.org/0000-0003-0268-2693 |
| Appears in Collections: | Dept of Health Sciences Embargoed Research Papers |
Files in This Item:
| File | Description | Size | Format | |
|---|---|---|---|---|
| ASPIRO PLSP Draft 5_04Apr2025 (1).pdf | Embargoed until publication | 617.25 kB | Adobe PDF | View/Open |
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