146 related articles for article (PubMed ID: 17570076)
21. Plasma Pharmacokinetics of Valanafusp Alpha, a Human Insulin Receptor Antibody-Iduronidase Fusion Protein, in Patients with Mucopolysaccharidosis Type I.
Pardridge WM; Boado RJ; Giugliani R; Schmidt M
BioDrugs; 2018 Apr; 32(2):169-176. PubMed ID: 29442294
[TBL] [Abstract][Full Text] [Related]
22. [The development of cognitive functions in children with Hurler phenotype mucopolysaccharidosis type I on enzyme replacement therapy with laronidase].
Biernacka M; Jakubowska-Winecka A; Tylki-Szymańska A
Pediatr Endocrinol Diabetes Metab; 2010; 16(4):249-54. PubMed ID: 21447265
[TBL] [Abstract][Full Text] [Related]
23. 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]
24. Growth in individuals with attenuated mucopolysaccharidosis type I during untreated and treated periods: Data from the MPS I registry.
Polgreen LE; Bay L; Clarke LA; Guffon N; Jones SA; Muenzer J; Flores AL; Wilson K; Viskochil D
Am J Med Genet A; 2022 Oct; 188(10):2941-2951. PubMed ID: 35869927
[TBL] [Abstract][Full Text] [Related]
25. 12 year follow up of enzyme-replacement therapy in two siblings with attenuated mucopolysaccharidosis I: the important role of early treatment.
Gabrielli O; Clarke LA; Ficcadenti A; Santoro L; Zampini L; Volpi N; Coppa GV
BMC Med Genet; 2016 Mar; 17():19. PubMed ID: 26965916
[TBL] [Abstract][Full Text] [Related]
26. Mucopolysaccharidosis type I: characterization of novel mutations affecting alpha-L-iduronidase activity.
Lee-Chen GJ; Lin SP; Tang YF; Chin YW
Clin Genet; 1999 Jul; 56(1):66-70. PubMed ID: 10466419
[TBL] [Abstract][Full Text] [Related]
27. 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]
28. Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector.
Di Domenico C; Villani GR; Di Napoli D; Reyero EG; Lombardo A; Naldini L; Di Natale P
Hum Gene Ther; 2005 Jan; 16(1):81-90. PubMed ID: 15703491
[TBL] [Abstract][Full Text] [Related]
29. Successful treatment of severe heart failure in an infant with Hurler syndrome.
Hirth A; Berg A; Greve G
J Inherit Metab Dis; 2007 Oct; 30(5):820. PubMed ID: 17768668
[TBL] [Abstract][Full Text] [Related]
30. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I.
Clarke LA; Wraith JE; Beck M; Kolodny EH; Pastores GM; Muenzer J; Rapoport DM; Berger KI; Sidman M; Kakkis ED; Cox GF
Pediatrics; 2009 Jan; 123(1):229-40. PubMed ID: 19117887
[TBL] [Abstract][Full Text] [Related]
31. An 8-year-old girl with a history of stiff and painful joints.
Raiman J; D'Aco K
Pediatr Ann; 2014 Aug; 43(8):307-9. PubMed ID: 25102484
[No Abstract] [Full Text] [Related]
32. Mapping of IDUA gene variants in Pakistani patients with mucopolysaccharidosis type 1.
Zahoor MY; Cheema HA; Ijaz S; Anjum MN; Ramzan K; Bhinder MA
J Pediatr Endocrinol Metab; 2019 Nov; 32(11):1221-1227. PubMed ID: 31473686
[TBL] [Abstract][Full Text] [Related]
33. Identification of a novel compound heterozygous IDUA mutation underlies Mucopolysaccharidoses type I in a Chinese pedigree.
Zhou YA; Li P; Zhang Y; Xiong Q; Li C; Zhao Z; Wang Y; Xiao H
Mol Genet Genomic Med; 2020 Jan; 8(1):e1058. PubMed ID: 31758674
[TBL] [Abstract][Full Text] [Related]
34. Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.
Dickson P; Peinovich M; McEntee M; Lester T; Le S; Krieger A; Manuel H; Jabagat C; Passage M; Kakkis ED
J Clin Invest; 2008 Aug; 118(8):2868-76. PubMed ID: 18654665
[TBL] [Abstract][Full Text] [Related]
35. Laronidase treatment of mucopolysaccharidosis I.
Wraith EJ; Hopwood JJ; Fuller M; Meikle PJ; Brooks DA
BioDrugs; 2005; 19(1):1-7. PubMed ID: 15691212
[TBL] [Abstract][Full Text] [Related]
36. Patient iPSC-derived neural stem cells exhibit phenotypes in concordance with the clinical severity of mucopolysaccharidosis I.
Swaroop M; Brooks MJ; Gieser L; Swaroop A; Zheng W
Hum Mol Genet; 2018 Oct; 27(20):3612-3626. PubMed ID: 30052969
[TBL] [Abstract][Full Text] [Related]
37. Mucopolysaccharidosis type I: Identification and characterization of mutations affecting alpha-L-iduronidase activity.
Lee-Chen GJ; Lin SP; Chen IS; Chang JH; Yang CW; Chin YW
J Formos Med Assoc; 2002 Jun; 101(6):425-8. PubMed ID: 12189649
[TBL] [Abstract][Full Text] [Related]
38. Postmortem studies on a patient with mucopolysaccharidosis type I: histopathological findings after one year of enzyme replacement therapy.
Yano S; Moseley K; Pavlova Z
J Inherit Metab Dis; 2009 Dec; 32 Suppl 1():S53-7. PubMed ID: 19308670
[TBL] [Abstract][Full Text] [Related]
39. Four novel mutations underlying mild or intermediate forms of alpha-L-iduronidase deficiency (MPS IS and MPS IH/S).
Tieu PT; Bach G; Matynia A; Hwang M; Neufeld EF
Hum Mutat; 1995; 6(1):55-9. PubMed ID: 7550232
[TBL] [Abstract][Full Text] [Related]
40. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years.
Sifuentes M; Doroshow R; Hoft R; Mason G; Walot I; Diament M; Okazaki S; Huff K; Cox GF; Swiedler SJ; Kakkis ED
Mol Genet Metab; 2007 Feb; 90(2):171-80. PubMed ID: 17011223
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
