Detalhe da pesquisa
1.
Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy.
Blood
; 133(12): 1378-1381, 2019 03 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-30635285
2.
Reduced-Toxicity (BuFlu) Conditioning Is Better Tolerated but Has a Higher Second Transplantation Rate Compared to Myeloablative Conditioning (BuCy) in Children with Inherited Metabolic Disorders.
Biol Blood Marrow Transplant
; 26(3): 486-492, 2020 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-31751770
3.
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
N Engl J Med
; 377(17): 1630-1638, 2017 10 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-28976817
4.
Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation.
Pediatr Res
; 87(1): 104-111, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31434105
5.
Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes.
Genet Med
; 21(11): 2552-2560, 2019 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-31019279
6.
Allele-Level HLA Matching Impacts Key Outcomes Following Umbilical Cord Blood Transplantation for Inherited Metabolic Disorders.
Biol Blood Marrow Transplant
; 23(1): 119-125, 2017 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-27989932
7.
Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience.
J Inherit Metab Dis
; 40(2): 271-280, 2017 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28054207
8.
The Frequency of Carpal Tunnel Syndrome in Hurler Syndrome After Peritransplant Enzyme Replacement Therapy: A Retrospective Comparison.
J Hand Surg Am
; 42(7): 573.e1-573.e8, 2017 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-28479223
9.
Impact of Graft-Recipient ABO Compatibility on Outcomes after Umbilical Cord Blood Transplant for Nonmalignant Disease.
Biol Blood Marrow Transplant
; 22(11): 2019-2024, 2016 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-27496217
10.
Outcomes after hematopoietic stem cell transplantation for children with I-cell disease.
Biol Blood Marrow Transplant
; 20(11): 1847-51, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-25016194
11.
Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report.
Blood
; 118(7): 1971-8, 2011 Aug 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-21586746
12.
Comparison of hypersensitivity reactions to PEG-asparaginase in children after intravenous and intramuscular administration.
J Pediatr Hematol Oncol
; 35(7): e283-6, 2013 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-23619117
13.
GVHD after haploidentical transplantation: a novel, MHC-defined rhesus macaque model identifies CD28- CD8+ T cells as a reservoir of breakthrough T-cell proliferation during costimulation blockade and sirolimus-based immunosuppression.
Blood
; 116(24): 5403-18, 2010 Dec 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-20833977
14.
Open-Label Pilot Study of Interferon Gamma-1b in Patients With Non-Infantile Osteopetrosis.
JBMR Plus
; 6(3): e10597, 2022 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-35309862
15.
Characterizing Immune-Mediated Cytopenias After Allogeneic Hematopoietic Cell Transplantation for Pediatric Nonmalignant Disorders.
Transplant Cell Ther
; 27(4): 316.e1-316.e8, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33836874
16.
Outcome After Cord Blood Transplantation Using Busulfan Pharmacokinetics-Targeted Myeloablative Conditioning for Hurler Syndrome.
Transplant Cell Ther
; 27(1): 91.e1-91.e4, 2021 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32961374
17.
Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy.
Neurology
; 95(5): e591-e600, 2020 08 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32616675
18.
Failure of intrathecal allogeneic mesenchymal stem cells to halt progressive demyelination in two boys with cerebral adrenoleukodystrophy.
Stem Cells Transl Med
; 9(5): 554-558, 2020 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-32020747
19.
Association between APOE4 and biomarkers in cerebral adrenoleukodystrophy.
Sci Rep
; 9(1): 7858, 2019 05 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-31133696
20.
Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes.
Sci Rep
; 9(1): 14105, 2019 Oct 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31575939