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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: mcfadyen a. 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.
Recommendations for the management of MPS IVA: 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: mcfadyen a. Orphanet J Rare Dis. 2019 Jun 13;14(1):137. doi: 10.1186/s13023-019-1074-9. Orphanet J Rare Dis. 2019. PMID: 31196221 Free PMC article.
What compassionate use means for gene therapies.
Chapman CR, Moch KI, McFadyen A, Kearns L, Watson T, Furlong P, Bateman-House A. Chapman CR, et al. Among authors: mcfadyen a. Nat Biotechnol. 2019 Apr;37(4):352-355. doi: 10.1038/s41587-019-0081-7. Nat Biotechnol. 2019. PMID: 30940936 No abstract available.
Fetal Therapies and Clinical Research: Beyond Risk and Benefit.
Shah LD, Lantos J, Hunt C, McFadyen A, Escandon R, Bateman-House A. Shah LD, et al. Among authors: mcfadyen a. Am J Bioeth. 2022 Mar;22(3):1-3. doi: 10.1080/15265161.2022.2035468. Epub 2022 Mar 8. Am J Bioeth. 2022. PMID: 38410955 No abstract available.
107 results