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5. Enzyme replacement therapy in feline mucopolysaccharidosis I. Kakkis ED; Schuchman E; He X; Wan Q; Kania S; Wiemelt S; Hasson CW; O'Malley T; Weil MA; Aguirre GA; Brown DE; Haskins ME Mol Genet Metab; 2001 Mar; 72(3):199-208. PubMed ID: 11243725 [TBL] [Abstract][Full Text] [Related]
6. Enzyme-replacement therapy in mucopolysaccharidosis I. Kakkis ED; Muenzer J; Tiller GE; Waber L; Belmont J; Passage M; Izykowski B; Phillips J; Doroshow R; Walot I; Hoft R; Neufeld EF N Engl J Med; 2001 Jan; 344(3):182-8. PubMed ID: 11172140 [TBL] [Abstract][Full Text] [Related]
7. Enzyme replacement therapy in mucopolysaccharidosis I: altered distribution and targeting of alpha-L-iduronidase in immunized rats. Turner CT; Hopwood JJ; Brooks DA Mol Genet Metab; 2000 Apr; 69(4):277-85. PubMed ID: 10870845 [TBL] [Abstract][Full Text] [Related]
8. [Hurler syndrome. Early diagnosis and successful enzyme replacement therapy: a new therapeutic approach. Case report]. Dupont C; El Hachem C; Harchaoui S; Ribault V; Amiour M; Guillot M; Maire I; Froissart R; Guffon-Fouilhoux N Arch Pediatr; 2008 Jan; 15(1):45-9. PubMed ID: 18162380 [TBL] [Abstract][Full Text] [Related]
9. Biomarker responses correlate with antibody status in mucopolysaccharidosis type I patients on long-term enzyme replacement therapy. Langereis EJ; van Vlies N; Church HJ; Geskus RB; Hollak CE; Jones SA; Kulik W; van Lenthe H; Mercer J; Schreider L; Tylee KL; Wagemans T; Wijburg FA; Bigger BW Mol Genet Metab; 2015 Feb; 114(2):129-37. PubMed ID: 25467058 [TBL] [Abstract][Full Text] [Related]
10. Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I. Kakkis E; McEntee M; Vogler C; Le S; Levy B; Belichenko P; Mobley W; Dickson P; Hanson S; Passage M Mol Genet Metab; 2004; 83(1-2):163-74. PubMed ID: 15464431 [TBL] [Abstract][Full Text] [Related]
11. Progressive eye pathology in mucopolysaccharidosis type I mice and effects of enzyme replacement therapy. Gonzalez EA; Visioli F; Pasqualim G; de Souza CFM; Marinho DR; Giugliani R; Matte U; Baldo G Clin Exp Ophthalmol; 2020 Apr; 48(3):334-342. PubMed ID: 31925897 [TBL] [Abstract][Full Text] [Related]
12. Ocular changes in patients with mucopolysaccharidosis I receiving enzyme replacement therapy: a 4-year experience. Pitz S; Ogun O; Bajbouj M; Arash L; Schulze-Frenking G; Beck M Arch Ophthalmol; 2007 Oct; 125(10):1353-6. PubMed ID: 17923542 [TBL] [Abstract][Full Text] [Related]
13. Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome. Cox-Brinkman J; Boelens JJ; Wraith JE; O'meara A; Veys P; Wijburg FA; Wulffraat N; Wynn RF Bone Marrow Transplant; 2006 Jul; 38(1):17-21. PubMed ID: 16715104 [TBL] [Abstract][Full Text] [Related]
14. Efficacy of laronidase therapy in patients with mucopolysaccharidosis type I who initiated enzyme replacement therapy in adult age. A systematic review and meta-analysis. Pérez-López J; Morales-Conejo M; López-Rodríguez M; Hermida-Ameijeiras Á; Moltó-Abad M Mol Genet Metab; 2017 Jun; 121(2):138-149. PubMed ID: 28410878 [TBL] [Abstract][Full Text] [Related]
15. Diagnosing lysosomal storage disorders: mucopolysaccharidosis type I. Johnson BA; Dajnoki A; Bodamer OA Curr Protoc Hum Genet; 2015 Jan; 84():17.17.1-17.17.8. PubMed ID: 25599668 [TBL] [Abstract][Full Text] [Related]
16. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Wraith JE; Beck M; Lane R; van der Ploeg A; Shapiro E; Xue Y; Kakkis ED; Guffon N Pediatrics; 2007 Jul; 120(1):e37-46. PubMed ID: 17606547 [TBL] [Abstract][Full Text] [Related]
17. Efficacy of recombinant human alpha-L-iduronidase (laronidase) on restricted range of motion of upper extremities in mucopolysaccharidosis type I patients. Tylki-Szymanska A; Marucha J; Jurecka A; Syczewska M; Czartoryska B J Inherit Metab Dis; 2010 Apr; 33(2):151-7. PubMed ID: 20217237 [TBL] [Abstract][Full Text] [Related]
18. Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I. Wang RY; Aminian A; McEntee MF; Kan SH; Simonaro CM; Lamanna WC; Lawrence R; Ellinwood NM; Guerra C; Le SQ; Dickson PI; Esko JD Mol Genet Metab; 2014 Aug; 112(4):286-93. PubMed ID: 24951454 [TBL] [Abstract][Full Text] [Related]
19. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). 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 J Pediatr; 2004 May; 144(5):581-8. PubMed ID: 15126990 [TBL] [Abstract][Full Text] [Related]
20. Effect of anti-laronidase antibodies on efficacy and safety of laronidase enzyme replacement therapy for MPS I: A comprehensive meta-analysis of pooled data from multiple studies. Xue Y; Richards SM; Mahmood A; Cox GF Mol Genet Metab; 2016 Apr; 117(4):419-26. PubMed ID: 26920513 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]