| Vazna A, Beesley C, Berna L, et al.
Mucopolysaccharidosis type I in 21 Czech and Slovak patients: Mutation analysis suggests a functional importance of C-terminus of the IDUA protein. [JOURNAL ARTICLE]
Am J Med Genet A 2009 Apr 24; 149A(5):965-974.
Abstract | Full Citation | Find Related Articles | | Aronovich EL, Bell JB, Khan SA, et al.
Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system. [Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't]
Mol Ther 2009 Jul; 17(7):1136-44.
Abstract | Full Citation | Publisher Full Text | Find Related Articles | | Ciron C, Cressant A, Roux F, et al.
Human alpha-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution. [JOURNAL ARTICLE]
Hum Gene Ther 2009 Feb 12.
Abstract | Full Citation | Publisher Full Text | Find Related Articles | | Wynn RF, Mercer J, Page J, et al.
Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. [Journal Article, Research Support, Non-U.S. Gov't]
J Pediatr 2009 Jan; 154(1):135-9.
Abstract | Full Citation | Publisher Full Text | Find Related Articles | | Clarke LA, Wraith JE, Beck M, et al.
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. [Journal Article]
Pediatrics 2009 Jan; 123(1):229-40.
Abstract | Full Citation | Publisher Full Text | Find Related Articles | | Blanchard S, Sadilek M, Scott CR, et al.
Tandem mass spectrometry for the direct assay of lysosomal enzymes in dried blood spots: application to screening newborns for mucopolysaccharidosis I. [Journal Article]
Clin Chem 2008 Dec; 54(12):2067-70.
Abstract | Full Citation | | Giugliani R, Rojas VM, Martins AM, et al.
A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. [Journal Article, Multicenter Study, Randomized Controlled Trial, Research Support, Non-U.S. Gov't]
Mol Genet Metab 2009 Jan; 96(1):13-9.
Abstract | Full Citation | Publisher Full Text | Find Related Articles | | Chen CH, Dellamaggiore KR, Ouellette CP, et al.
Aptamer-based endocytosis of a lysosomal enzyme. [Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't]
Proc Natl Acad Sci U S A 2008 Oct 14; 105(41):15908-13.
Abstract | Full Citation | Publisher Full Text | Find Related Articles | | Munoz-Rojas MV, Vieira T, Costa R, et al.
Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression. [JOURNAL ARTICLE]
Am J Med Genet A 2008 Sep 15.
Abstract | Full Citation | | Tsukimura T, Tajima Y, Kawashima I, et al.
Uptake of a Recombinant Human alpha-L-Iduronidase (laronidase) by Cultured Fibroblasts and Osteoblasts. [JOURNAL ARTICLE]
Biol Pharm Bull 2008; 31(9):1691-1695.
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