| Title: |
An algorithm for discontinuing mechanical ventilation in boys with x-linked myotubular myopathy after positive response to gene therapy: the ASPIRO experience |
| Authors: |
Graham, RJ; Amin, R; Demirel, N; Edel, L; Lilien, C; MacBean, V; Rafferty, GF; Sawnani, H; Schön, C; Smith, BK; Syed, F; Sarazen, M; Prasad, S; Rico, S; Perez, GF |
| Publisher Information: |
BioMed Central |
| Publication Year: |
2026 |
| Collection: |
Oxford University Research Archive (ORA) |
| Description: |
Availability of data and materials: Not applicable.The ASPIRO trial was sponsored by Astellas Gene Therapies (formerly Audentes Therapeutics) and was the impetus for development of the algorithm for weaning ventilator-dependent children with XLMTM off of ventilator support. Medical writing support from Laurie LaRusso, MS, ELS, of Chestnut Medical Communications was paid for by Astellas Gene Therapies |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| DOI: |
10.1186/s12931-024-02966-0 |
| Availability: |
https://doi.org/10.1186/s12931-024-02966-0; https://ora.ox.ac.uk/objects/uuid:5e7e2100-7b27-46d3-a0cf-d86da44a1067 |
| Rights: |
info:eu-repo/semantics/openAccess ; CC Attribution (CC BY) |
| Accession Number: |
edsbas.5E8FC28E |
| Database: |
BASE |