227 related articles for article (PubMed ID: 28983455)
1. 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]
2. Early enzyme replacement therapy enables a successful hematopoietic stem cell transplantation in mucopolysaccharidosis type IH: Divergent clinical outcomes in two Japanese siblings.
Yamazaki N; Kosuga M; Kida K; Takei G; Fukuhara Y; Matsumoto H; Senda M; Honda A; Ishiguro A; Koike T; Yabe H; Okuyama T
Brain Dev; 2019 Jun; 41(6):546-550. PubMed ID: 30755342
[TBL] [Abstract][Full Text] [Related]
3. 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]
4. Mucopolysaccharidosis type I.
Wraith JE; Jones S
Pediatr Endocrinol Rev; 2014 Sep; 12 Suppl 1():102-6. PubMed ID: 25345091
[TBL] [Abstract][Full Text] [Related]
5. Hematopoietic cell transplantation for Mucopolysaccharidosis I in the presence of decreased cardiac function.
Pillai NR; Elsbecker SA; Gupta AO; Lund TC; Orchard PJ; Braunlin E
Mol Genet Metab; 2023 Nov; 140(3):107669. PubMed ID: 37542767
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. Timing is everything: Clinical courses of Hunter syndrome associated with age at initiation of therapy in a sibling pair.
Grant N; Sohn YB; Ellinwood NM; Okenfuss E; Mendelsohn BA; Lynch LE; Braunlin EA; Harmatz PR; Eisengart JB
Mol Genet Metab Rep; 2022 Mar; 30():100845. PubMed ID: 35242576
[TBL] [Abstract][Full Text] [Related]
8. Residual α-L-iduronidase activity in fibroblasts of mild to severe Mucopolysaccharidosis type I patients.
Oussoren E; Keulemans J; van Diggelen OP; Oemardien LF; Timmermans RG; van der Ploeg AT; Ruijter GJ
Mol Genet Metab; 2013 Aug; 109(4):377-81. PubMed ID: 23786846
[TBL] [Abstract][Full Text] [Related]
9. The natural history of neurocognition in MPS disorders: A review.
Shapiro EG; Eisengart JB
Mol Genet Metab; 2021 May; 133(1):8-34. PubMed ID: 33741271
[TBL] [Abstract][Full Text] [Related]
10. alpha-L-iduronidase therapy for mucopolysaccharidosis type I.
Tolar J; Orchard PJ
Biologics; 2008 Dec; 2(4):743-51. PubMed ID: 19707455
[TBL] [Abstract][Full Text] [Related]
11. Hematopoietic cell transplantation for severe MPS I in the first six months of life: The heart of the matter.
Braunlin E; Miettunen K; Lund T; Luquette M; Orchard P
Mol Genet Metab; 2019 Feb; 126(2):117-120. PubMed ID: 30503158
[TBL] [Abstract][Full Text] [Related]
12. Cerebral magnetic resonance findings during enzyme replacement therapy in mucopolysaccharidosis.
Matsubara Y; Miyazaki O; Kosuga M; Okuyama T; Nosaka S
Pediatr Radiol; 2017 Nov; 47(12):1659-1669. PubMed ID: 28733853
[TBL] [Abstract][Full Text] [Related]
13. "Growth patterns in children with mucopolysaccharidosis type I-Hurler after hematopoietic stem cell transplantation: Comparison with untreated patients".
Cattoni A; Chiaraluce S; Gasperini S; Molinari S; Biondi A; Rovelli A; Parini R
Mol Genet Metab Rep; 2021 Sep; 28():100787. PubMed ID: 34408967
[TBL] [Abstract][Full Text] [Related]
14. Effects of neonatal enzyme replacement therapy and simvastatin treatment on cervical spine disease in mucopolysaccharidosis I dogs.
Chiaro JA; O'Donnell P; Shore EM; Malhotra NR; Ponder KP; Haskins ME; Smith LJ
J Bone Miner Res; 2014 Dec; 29(12):2610-7. PubMed ID: 24898323
[TBL] [Abstract][Full Text] [Related]
15. Enzyme replacement therapy: efficacy and limitations.
Concolino D; Deodato F; Parini R
Ital J Pediatr; 2018 Nov; 44(Suppl 2):120. PubMed ID: 30442189
[TBL] [Abstract][Full Text] [Related]
16. Scheie syndrome: enzyme replacement therapy does not prevent progression of cervical myelopathy due to spinal cord compression.
Illsinger S; Lücke T; Hartmann H; Mengel E; Müller-Forell W; Donnerstag F; Das AM
J Inherit Metab Dis; 2009 Dec; 32 Suppl 1():S321-5. PubMed ID: 19894140
[TBL] [Abstract][Full Text] [Related]
17. Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment.
Shapiro EG; Nestrasil I; Rudser K; Delaney K; Kovac V; Ahmed A; Yund B; Orchard PJ; Eisengart J; Niklason GR; Raiman J; Mamak E; Cowan MJ; Bailey-Olson M; Harmatz P; Shankar SP; Cagle S; Ali N; Steiner RD; Wozniak J; Lim KO; Whitley CB
Mol Genet Metab; 2015; 116(1-2):61-8. PubMed ID: 26095521
[TBL] [Abstract][Full Text] [Related]
18. The evolution of pulmonary function in childhood onset Mucopolysaccharidosis type I.
Broomfield A; Sims J; Mercer J; Hensman P; Ghosh A; Tylee K; Stepien KM; Oldham A; Prathivadi Bhayankaram N; Wynn R; Wright NB; Jones SA; Wilkinson S
Mol Genet Metab; 2021 Feb; 132(2):94-99. PubMed ID: 32713717
[TBL] [Abstract][Full Text] [Related]
19. Natural history of craniovertebral abnormalities in a single-center study in 54 patients with Hurler syndrome.
Huang S; Nascene DR; Shanley R; Pena-Pino I; Lund TC; Gupta AO; Orchard PJ; Sandoval-Garcia C
J Neurosurg Pediatr; 2024 Jun; 33(6):574-582. PubMed ID: 38489810
[TBL] [Abstract][Full Text] [Related]
20. Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI.
Furujo M; Kubo T; Kosuga M; Okuyama T
Mol Genet Metab; 2011 Dec; 104(4):597-602. PubMed ID: 21930407
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
