BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

185 related articles for article (PubMed ID: 22974573)

  • 1. Enzyme replacement is associated with better cognitive outcomes after transplant in Hurler syndrome.
    Eisengart JB; Rudser KD; Tolar J; Orchard PJ; Kivisto T; Ziegler RS; Whitley CB; Shapiro EG
    J Pediatr; 2013 Feb; 162(2):375-80.e1. PubMed ID: 22974573
    [TBL] [Abstract][Full Text] [Related]  

  • 2. The Frequency of Carpal Tunnel Syndrome in Hurler Syndrome After Peritransplant Enzyme Replacement Therapy: A Retrospective Comparison.
    Wyffels ML; Orchard PJ; Shanley RM; Miller WP; Van Heest AE
    J Hand Surg Am; 2017 Jul; 42(7):573.e1-573.e8. PubMed ID: 28479223
    [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. Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes.
    Lund TC; Miller WP; Liao AY; Tolar J; Shanley R; Pasquali M; Sando N; Bigger BW; Polgreen LE; Orchard PJ
    Sci Rep; 2019 Oct; 9(1):14105. PubMed ID: 31575939
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease.
    Potter JE; Petts G; Ghosh A; White FJ; Kinsella JL; Hughes S; Roberts J; Hodgkinson A; Brammeier K; Church H; Merrigan C; Hughes J; Evans P; Campbell H; Bonney D; Newman WG; Bigger BW; Broomfield A; Jones SA; Wynn RF
    Orphanet J Rare Dis; 2021 May; 16(1):235. PubMed ID: 34020687
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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]  

  • 8. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome.
    Grewal SS; Wynn R; Abdenur JE; Burton BK; Gharib M; Haase C; Hayashi RJ; Shenoy S; Sillence D; Tiller GE; Dudek ME; van Royen-Kerkhof A; Wraith JE; Woodard P; Young GA; Wulffraat N; Whitley CB; Peters C
    Genet Med; 2005 Feb; 7(2):143-6. PubMed ID: 15714083
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome.
    Bijarnia S; Shaw P; Vimpani A; Smith R; Pacey V; O'Grady H; Christodoulou J; Sillence D
    J Paediatr Child Health; 2009; 45(7-8):469-72. PubMed ID: 19712183
    [TBL] [Abstract][Full Text] [Related]  

  • 10. 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]  

  • 11. 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]  

  • 12. 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]  

  • 13. 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]  

  • 14. 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]  

  • 15. 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]  

  • 16. 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]  

  • 17. 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]  

  • 18. Progression of organ manifestations upon enzyme replacement therapy in a patient with mucopolysaccharidosis type I/Hurler.
    Mercimek-Mahmutoglu S; Reilly C; Human D; Waters PJ; Stoeckler-Ipsiroglu S
    World J Pediatr; 2009 Nov; 5(4):319-21. PubMed ID: 19911152
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Combination of enzyme replacement and hematopoietic stem cell transplantation as therapy for Hurler syndrome.
    Tolar J; Grewal SS; Bjoraker KJ; Whitley CB; Shapiro EG; Charnas L; Orchard PJ
    Bone Marrow Transplant; 2008 Mar; 41(6):531-5. PubMed ID: 18037941
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study.
    Aldenhoven M; Wynn RF; Orchard PJ; O'Meara A; Veys P; Fischer A; Valayannopoulos V; Neven B; Rovelli A; Prasad VK; Tolar J; Allewelt H; Jones SA; Parini R; Renard M; Bordon V; Wulffraat NM; de Koning TJ; Shapiro EG; Kurtzberg J; Boelens JJ
    Blood; 2015 Mar; 125(13):2164-72. PubMed ID: 25624320
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 10.