REFERENCES
- Ito S, Barrett AJ. Gaucher's disease–a reappraisal of hematopoietic stem cell transplantation. Pediatr Hematol Oncol. 2013;30(2):61–70.
- Treiber A, Morand O, Clozel M. The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat. Xenobiotica. 2007;37(3):298–314.
- Schiffmann R, Fitzgibbon EJ, Harris C, Randomized, controlled trial of miglustat in Gaucher's disease type 3. Ann Neurol. 2008;64(5):514–522.
- Zimran A. How I treat Gaucher disease. Blood. 2011;118(6):1463–1471.
- Shayman JA. Eliglustat tartrate: glucosylceramide synthase inhibitor treatment of type 1 Gaucher disease. Drugs Future. 2010;35(8):613–620.
- Hsieh MM, Kang EM, Fitzhugh CD, Allogeneic hematopoietic stem-cell transplantation for sickle cell disease. N Engl J Med. 2009;361(24):2309–2317.
- Hsieh MM, Fitzhugh CD, Tisdale JF. Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now. Blood. 2011;118(5):1197–1207.
- Kraoua I, Sedel F, Caillaud C, A French experience of type 3 Gaucher disease: phenotypic diversity and neurological outcome of 10 patients. Brain Dev. 2011;33(2):131–139.
- Staba SL, Escolar ML, Poe M, Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004;350(19):1960–1969.