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Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.
Ellison S, Buckland K, Learmonth Y, Day V, Kalra S, Howe L, Roman-Rodriguez FJ, Bonafont J, Booth L, Holley R, Smythe J, Jones S, Thrasher A, Booth C, Bigger BW. Ellison S, et al. Mol Ther Methods Clin Dev. 2024 May 21;32(2):101271. doi: 10.1016/j.omtm.2024.101271. eCollection 2024 Jun 13. Mol Ther Methods Clin Dev. 2024. PMID: 38946936 Free PMC article.
Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance.
Akyol MU, Alden TD, Amartino H, Ashworth J, Belani K, Berger KI, Borgo A, Braunlin E, Eto Y, Gold JI, Jester A, Jones SA, Karsli C, Mackenzie W, Marinho DR, McFadyen A, McGill J, Mitchell JJ, Muenzer J, Okuyama T, Orchard PJ, Stevens B, Thomas S, Walker R, Wynn R, Giugliani R, Harmatz P, Hendriksz C, Scarpa M; MPS Consensus Programme Steering Committee; MPS Consensus Programme Co-Chairs. Akyol MU, et al. Among authors: jones sa. Orphanet J Rare Dis. 2019 May 29;14(1):118. doi: 10.1186/s13023-019-1080-y. Orphanet J Rare Dis. 2019. PMID: 31142378 Free PMC article.
Evaluation of early treatment with intravenous idursulfase and intrathecal idursulfase-IT on cognitive function in siblings with neuronopathic mucopolysaccharidosis II.
Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Rust S, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D. Muenzer J, et al. Among authors: jones sa. J Inherit Metab Dis. 2024 Sep 9. doi: 10.1002/jimd.12790. Online ahead of print. J Inherit Metab Dis. 2024. PMID: 39252529
932 results