191 related articles for article (PubMed ID: 35304037)
41. Failure to shorten the diagnostic delay in two ultra-orphan diseases (mucopolysaccharidosis types I and III): potential causes and implications.
Kuiper GA; Meijer OLM; Langereis EJ; Wijburg FA
Orphanet J Rare Dis; 2018 Jan; 13(1):2. PubMed ID: 29310675
[TBL] [Abstract][Full Text] [Related]
42. Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation.
Polgreen LE; Lund TC; Braunlin E; Tolar J; Miller BS; Fung E; Whitley CB; Eisengart JB; Northrop E; Rudser K; Miller WP; Orchard PJ
Pediatr Res; 2020 Jan; 87(1):104-111. PubMed ID: 31434105
[TBL] [Abstract][Full Text] [Related]
43. An exploratory study of brain function and structure in mucopolysaccharidosis type I: long term observations following hematopoietic cell transplantation (HCT).
Shapiro E; Guler OE; Rudser K; Delaney K; Bjoraker K; Whitley C; Tolar J; Orchard P; Provenzale J; Thomas KM
Mol Genet Metab; 2012 Sep; 107(1-2):116-21. PubMed ID: 22867884
[TBL] [Abstract][Full Text] [Related]
44. Mucopolysaccharidoses I and II: Brief Review of Therapeutic Options and Supportive/Palliative Therapies.
Nan H; Park C; Maeng S
Biomed Res Int; 2020; 2020():2408402. PubMed ID: 33344633
[No Abstract] [Full Text] [Related]
45. Ability change across multiple domains in mucopolysaccharidosis (Sanfilippo syndrome) type IIIA.
Shapiro EG; Eisengart JB; Whiteman D; Whitley CB
Mol Genet Metab; 2024 Feb; 141(2):108110. PubMed ID: 38151384
[TBL] [Abstract][Full Text] [Related]
46. An online survey on burden of illness among families with post-stem cell transplant mucopolysaccharidosis type I children in the United States.
Conner T; Cook F; Fernandez V; Rascati K; Rangel-Miller V
Orphanet J Rare Dis; 2019 Feb; 14(1):48. PubMed ID: 30777108
[TBL] [Abstract][Full Text] [Related]
47. Carpal tunnel syndrome in mucopolysaccharidosis I: a registry-based cohort study.
Viskochil D; Muenzer J; Guffon N; Garin C; Munoz-Rojas MV; Moy KA; Hutchinson DT
Dev Med Child Neurol; 2017 Dec; 59(12):1269-1275. PubMed ID: 28892147
[TBL] [Abstract][Full Text] [Related]
48. Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure.
Langereis EJ; Borgo A; Crushell E; Harmatz PR; van Hasselt PM; Jones SA; Kelly PM; Lampe C; van der Lee JH; Odent T; Sakkers R; Scarpa M; Schafroth MU; Struijs PA; Valayannopoulos V; White KK; Wijburg FA
Orphanet J Rare Dis; 2013 Oct; 8():155. PubMed ID: 24088413
[TBL] [Abstract][Full Text] [Related]
49. Improvement in time to treatment, but not time to diagnosis, in patients with mucopolysaccharidosis type I.
Giugliani R; Muschol N; Keenan HA; Dant M; Muenzer J
Arch Dis Child; 2021 Jul; 106(7):674-679. PubMed ID: 33139350
[TBL] [Abstract][Full Text] [Related]
50. Long-term cognitive and somatic outcomes of enzyme replacement therapy in untransplanted Hurler syndrome.
Eisengart JB; Jarnes J; Ahmed A; Nestrasil I; Ziegler R; Delaney K; Shapiro E; Whitley C
Mol Genet Metab Rep; 2017 Dec; 13():64-68. PubMed ID: 28983455
[TBL] [Abstract][Full Text] [Related]
51. [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]
52. Growth patterns in children with mucopolysaccharidosis I and II.
Różdżyńska-Świątkowska A; Jurecka A; Cieślik J; Tylki-Szymańska A
World J Pediatr; 2015 Aug; 11(3):226-31. PubMed ID: 25410665
[TBL] [Abstract][Full Text] [Related]
53. Psychosocial outcomes of bone marrow transplant for individuals affected by Mucopolysaccharidosis I Hurler Disease: patient social competency.
Pitt C; Lavery C; Wager N
Child Care Health Dev; 2009 Mar; 35(2):271-80. PubMed ID: 19228160
[TBL] [Abstract][Full Text] [Related]
54. Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison.
Eisengart JB; Rudser KD; Xue Y; Orchard P; Miller W; Lund T; Van der Ploeg A; Mercer J; Jones S; Mengel KE; Gökce S; Guffon N; Giugliani R; de Souza CFM; Shapiro EG; Whitley CB
Genet Med; 2018 Nov; 20(11):1423-1429. PubMed ID: 29517765
[TBL] [Abstract][Full Text] [Related]
55. Retrospective chart review of urinary glycosaminoglycan excretion and long-term clinical outcomes of enzyme replacement therapy in patients with mucopolysaccharidoses.
Jones SA; Marsden D; Koutsoukos T; Sniadecki J; Tylee K; Phillippo S; Kakkis E
Mol Genet Metab; 2020 Aug; 130(4):255-261. PubMed ID: 32563631
[TBL] [Abstract][Full Text] [Related]
56. Early Neonatal Cardiac Phenotype in Hurler Syndrome: Case Report and Literature Review.
Pillai NR; Ahmed A; Vanyo T; Whitley CB
Genes (Basel); 2022 Jul; 13(8):. PubMed ID: 35893030
[TBL] [Abstract][Full Text] [Related]
57. Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome.
Shapiro EG; Whitley CB; Eisengart JB
Orphanet J Rare Dis; 2018 May; 13(1):76. PubMed ID: 29751845
[TBL] [Abstract][Full Text] [Related]
58. Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines.
Aldenhoven M; Jones SA; Bonney D; Borrill RE; Coussons M; Mercer J; Bierings MB; Versluys B; van Hasselt PM; Wijburg FA; van der Ploeg AT; Wynn RF; Boelens JJ
Biol Blood Marrow Transplant; 2015 Jun; 21(6):1106-9. PubMed ID: 25708213
[TBL] [Abstract][Full Text] [Related]
59. Changes in Corneal Clouding Over Time in Patients With Mucopolysaccharidosis.
McGrath O; Sornalingam K; Aslam T; Ashworth J
Cornea; 2023 Aug; 42(8):992-999. PubMed ID: 36857777
[TBL] [Abstract][Full Text] [Related]
60. Health-related quality of life in children and adolescents who have a diagnosis of attention-deficit/hyperactivity disorder.
Klassen AF; Miller A; Fine S
Pediatrics; 2004 Nov; 114(5):e541-7. PubMed ID: 15520087
[TBL] [Abstract][Full Text] [Related]
[Previous] [Next] [New Search]
