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Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.
Dickson PI, Kaitila I, Harmatz P, Mlikotic A, Chen AH, Victoroff A, Passage MB, Madden J, Le SQ, Naylor DE; Mucopolysaccharidosis I Intrathecal Research Collaborative. Dickson PI, et al. Among authors: harmatz p. Mol Genet Metab. 2015 Sep-Oct;116(1-2):69-74. doi: 10.1016/j.ymgme.2015.07.005. Epub 2015 Jul 26. Mol Genet Metab. 2015. PMID: 26260077 Free PMC article. Clinical Trial.
Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload.
Fung EB, Harmatz P, Milet M, Ballas SK, De Castro L, Hagar W, Owen W, Olivieri N, Smith-Whitley K, Darbari D, Wang W, Vichinsky E; Multi-Center Study of Iron Overload Research Group. Fung EB, et al. Among authors: harmatz p. Am J Hematol. 2007 Apr;82(4):255-65. doi: 10.1002/ajh.20809. Am J Hematol. 2007. PMID: 17094096 Free article.
Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study.
Chen AH, Harmatz P, Nestrasil I, Eisengart JB, King KE, Rudser K, Kaizer AM, Svatkova A, Wakumoto A, Le SQ, Madden J, Young S, Zhang H, Polgreen LE, Dickson PI. Chen AH, et al. Among authors: harmatz p. Mol Genet Metab. 2020 Feb;129(2):80-90. doi: 10.1016/j.ymgme.2019.11.007. Epub 2019 Nov 30. Mol Genet Metab. 2020. PMID: 31839529 Free PMC article. Clinical Trial.
Increased leucocyte apoptosis in transfused β-thalassaemia patients.
Walter PB, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Grady RW, Vichinsky E, Olivieri N, Trachtenberg F, Alberti D, Fung E, Ames B, Higa A, Harmatz P; Thalassemia Clinical Research Network. Walter PB, et al. Among authors: harmatz p. Br J Haematol. 2013 Feb;160(3):399-403. doi: 10.1111/bjh.12076. Epub 2012 Dec 6. Br J Haematol. 2013. PMID: 23216540 Free PMC article.
231 results