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MPS I (clinicaltrials.gov)
A list of ongoing clinical studies for which patients may be eligible. The list includes registered studies that are closed and may have links to their published outcomes.
MPS I Registry
An ongoing, observational database that tracks natural history and outcomes of patients with MPS I. The Registry was initiated worldwide in April 2003 as an international observational program sponsored by BioMarin/Genzyme LLC and administered by Genzyme Corporation. Registration is voluntary, free, and confidential.
|Content Last Updated:||11/2011|
Cox-Brinkman J, Boelens JJ, Wraith JE, O'meara A, Veys P, Wijburg FA, Wulffraat N, Wynn RF.
Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome.
Bone Marrow Transplant. 2006;38(1):17-21. PubMed abstract
Gassas A, Sung L, Doyle JJ, Clarke JT, Saunders EF.
Life-threatening pulmonary hemorrhages post bone marrow transplantation in Hurler syndrome. Report of three cases and review of the literature.
Bone Marrow Transplant. 2003;32(2):213-5. PubMed abstract
Muenzer J, Fisher A.
Advances in the treatment of mucopolysaccharidosis type I.
N Engl J Med. 2004;350(19):1932-4. PubMed abstract
Souillet G, Guffon N, Maire I, Pujol M, Taylor P, Sevin F, Bleyzac N, Mulier C, Durin A, Kebaili K, Galambrun C, Bertrand
Y, Froissart R, Dorche C, Gebuhrer L, Garin C, Berard J, Guibaud P.
Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources.
Bone Marrow Transplant. 2003;31(12):1105-17. PubMed abstract
Weisstein JS, Delgado E, Steinbach LS, Hart K, Packman S.
Musculoskeletal manifestations of Hurler syndrome: long-term follow-up after bone marrow transplantation.
J Pediatr Orthop. 2004;24(1):97-101. PubMed abstract
The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I.
Expert Opin Pharmacother. 2005;6(3):489-506. PubMed abstract
Wraith JE, Clarke LA, Beck M, Kolodny EH, Pastores GM, Muenzer J, Rapoport DM, Berger KI, Swiedler SJ, Kakkis ED, Braakman
T, Chadbourne E, Walton-Bowen K, Cox GF.
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).
J Pediatr. 2004;144(5):581-8. PubMed abstract