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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: orchard pj. 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.
Pre-transplant risk factors affecting outcome in Hurler syndrome.
Orchard PJ, Milla C, Braunlin E, DeFor T, Bjoraker K, Blazar BR, Peters C, Wagner J, Tolar J. Orchard PJ, et al. Bone Marrow Transplant. 2010 Jul;45(7):1239-46. doi: 10.1038/bmt.2009.319. Epub 2009 Nov 9. Bone Marrow Transplant. 2010. PMID: 19898501
Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.
Aldenhoven M, Wynn RF, Orchard PJ, O'Meara A, Veys P, Fischer A, Valayannopoulos V, Neven B, Rovelli A, Prasad VK, Tolar J, Allewelt H, Jones SA, Parini R, Renard M, Bordon V, Wulffraat NM, de Koning TJ, Shapiro EG, Kurtzberg J, Boelens JJ. Aldenhoven M, et al. Among authors: orchard pj. Blood. 2015 Mar 26;125(13):2164-72. doi: 10.1182/blood-2014-11-608075. Epub 2015 Jan 26. Blood. 2015. PMID: 25624320 Free article.
Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment.
Shapiro EG, Nestrasil I, Rudser K, Delaney K, Kovac V, Ahmed A, Yund B, Orchard PJ, Eisengart J, Niklason GR, Raiman J, Mamak E, Cowan MJ, Bailey-Olson M, Harmatz P, Shankar SP, Cagle S, Ali N, Steiner RD, Wozniak J, Lim KO, Whitley CB. Shapiro EG, et al. Among authors: orchard pj. Mol Genet Metab. 2015 Sep-Oct;116(1-2):61-8. doi: 10.1016/j.ymgme.2015.06.002. Epub 2015 Jun 17. Mol Genet Metab. 2015. PMID: 26095521 Free PMC article. Clinical Trial.
Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation.
Kunin-Batson AS, Shapiro EG, Rudser KD, Lavery CA, Bjoraker KJ, Jones SA, Wynn RF, Vellodi A, Tolar J, Orchard PJ, Wraith JE. Kunin-Batson AS, et al. Among authors: orchard pj. JIMD Rep. 2016;29:95-102. doi: 10.1007/8904_2015_521. Epub 2016 Jan 30. JIMD Rep. 2016. PMID: 26825088 Free PMC article.
228 results