Clinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world
Author:
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
http://link.springer.com/content/pdf/10.1007/s10545-011-9336-2
Reference22 articles.
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2. Boelens JJ, Wynn RF, O'Meara A et al (2007) Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant 40:225–233
3. Clarke L, Wraith JE, Beck M et al (2009) Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics 123:229–240
4. Cox-Brinkman J, Boelens JJ, Wraith JE et al (2006) Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Bone Marrow Transplant 38:17–21
5. Dierenfeld AD, McEntee MF, Vogler CA, et al. (2010) Replacing the enzyme a-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I. Sci Transl Med 2: 60ra89
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