147 related articles for article (PubMed ID: 27991992)
1. Neurological outcomes after hematopoietic stem cell transplantation for cerebral X-linked adrenoleukodystrophy, late onset metachromatic leukodystrophy and Hurler syndrome.
Saute JA; Souza CF; Poswar FO; Donis KC; Campos LG; Deyl AV; Burin MG; Vargas CR; Matte UD; Giugliani R; Saraiva-Pereira ML; Vedolin LM; Gregianin LJ; Jardim LB
Arq Neuropsiquiatr; 2016 Dec; 74(12):953-966. PubMed ID: 27991992
[TBL] [Abstract][Full Text] [Related]
2. Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Juvenile Metachromatic Leukodystrophy Compared With Nontransplanted Control Patients.
Groeschel S; Kühl JS; Bley AE; Kehrer C; Weschke B; Döring M; Böhringer J; Schrum J; Santer R; Kohlschütter A; Krägeloh-Mann I; Müller I
JAMA Neurol; 2016 Sep; 73(9):1133-40. PubMed ID: 27400410
[TBL] [Abstract][Full Text] [Related]
3. Hematopoietic stem cell gene therapy in Hurler syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy and X-adrenoleukodystrophy.
Cartier N; Aubourg P
Curr Opin Mol Ther; 2008 Oct; 10(5):471-8. PubMed ID: 18830923
[TBL] [Abstract][Full Text] [Related]
4. The future for treatment by bone marrow transplantation for adrenoleukodystrophy, metachromatic leukodystrophy, globoid cell leukodystrophy and Hurler syndrome.
Krivit W; Lockman LA; Watkins PA; Hirsch J; Shapiro EG
J Inherit Metab Dis; 1995; 18(4):398-412. PubMed ID: 7494399
[TBL] [Abstract][Full Text] [Related]
5. Late Mortality after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism: A Report from the Blood or Marrow Transplant Survivor Study-2 (BMTSS-2).
Wadhwa A; Chen Y; Holmqvist A; Wu J; Ness E; Parman M; Kung M; Hageman L; Francisco L; Braunlin E; Miller W; Lund T; Armenian S; Arora M; Orchard P; Bhatia S
Biol Blood Marrow Transplant; 2019 Feb; 25(2):328-334. PubMed ID: 30292746
[TBL] [Abstract][Full Text] [Related]
6. Allogeneic mesenchymal stem cell infusion for treatment of metachromatic leukodystrophy (MLD) and Hurler syndrome (MPS-IH).
Koç ON; Day J; Nieder M; Gerson SL; Lazarus HM; Krivit W
Bone Marrow Transplant; 2002 Aug; 30(4):215-22. PubMed ID: 12203137
[TBL] [Abstract][Full Text] [Related]
7. Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report.
Boucher AA; Miller W; Shanley R; Ziegler R; Lund T; Raymond G; Orchard PJ
Orphanet J Rare Dis; 2015 Aug; 10():94. PubMed ID: 26245762
[TBL] [Abstract][Full Text] [Related]
8. Long-term adaptive functioning outcomes of children with inherited metabolic and genetic diseases treated with hematopoietic stem cell transplantation in a single large pediatric center: parents' perspective.
Gassas A; Raiman J; White L; Schechter T; Clarke J; Doyle J
J Pediatr Hematol Oncol; 2011 Apr; 33(3):216-20. PubMed ID: 21336168
[TBL] [Abstract][Full Text] [Related]
9. Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation.
Raymond GV; Aubourg P; Paker A; Escolar M; Fischer A; Blanche S; Baruchel A; Dalle JH; Michel G; Prasad V; Miller W; Paadre S; Balser J; Kurtzberg J; Nascene DR; Orchard PJ; Lund T
Biol Blood Marrow Transplant; 2019 Mar; 25(3):538-548. PubMed ID: 30292747
[TBL] [Abstract][Full Text] [Related]
10. Hematopoietic Stem Cell Transplantation for Children With Inborn Errors of Metabolism: Single Center Experience Over Two Decades.
Swaminathan VV; Meena S; Varla H; Chandar R; Jayakumar I; Ramakrishnan B; Uppuluri R; Raj R
Indian Pediatr; 2022 Sep; 59(9):699-702. PubMed ID: 35762023
[TBL] [Abstract][Full Text] [Related]
11. Neurodevelopmental outcomes of umbilical cord blood transplantation in metachromatic leukodystrophy.
Martin HR; Poe MD; Provenzale JM; Kurtzberg J; Mendizabal A; Escolar ML
Biol Blood Marrow Transplant; 2013 Apr; 19(4):616-24. PubMed ID: 23348427
[TBL] [Abstract][Full Text] [Related]
12. The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome).
Miebach E; Church H; Cooper A; Mercer J; Tylee K; Wynn RF; Wraith JE
J Inherit Metab Dis; 2011 Jun; 34(3):755-61. PubMed ID: 21416193
[TBL] [Abstract][Full Text] [Related]
13. Neurocognitive Trajectory of Boys Who Received a Hematopoietic Stem Cell Transplant at an Early Stage of Childhood Cerebral Adrenoleukodystrophy.
Pierpont EI; Eisengart JB; Shanley R; Nascene D; Raymond GV; Shapiro EG; Ziegler RS; Orchard PJ; Miller WP
JAMA Neurol; 2017 Jun; 74(6):710-717. PubMed ID: 28418523
[TBL] [Abstract][Full Text] [Related]
14. Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation.
Polgreen LE; Tolar J; Plog M; Himes JH; Orchard PJ; Whitley CB; Miller BS; Petryk A
Bone Marrow Transplant; 2008 Jun; 41(12):1005-11. PubMed ID: 18278070
[TBL] [Abstract][Full Text] [Related]
15. Potential Risks to Stable Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children With Cerebral X-linked Adrenoleukodystrophy.
Kühl JS; Kupper J; Baqué H; Ebell W; Gärtner J; Korenke C; Spors B; Steffen IG; Strauss G; Voigt S; Weschke B; Weddige A; Köhler W; Steinfeld R
JAMA Netw Open; 2018 Jul; 1(3):e180769. PubMed ID: 30646031
[TBL] [Abstract][Full Text] [Related]
16. Transplant outcomes in leukodystrophies.
Orchard PJ; Tolar J
Semin Hematol; 2010 Jan; 47(1):70-8. PubMed ID: 20109614
[TBL] [Abstract][Full Text] [Related]
17. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial.
Sessa M; Lorioli L; Fumagalli F; Acquati S; Redaelli D; Baldoli C; Canale S; Lopez ID; Morena F; Calabria A; Fiori R; Silvani P; Rancoita PM; Gabaldo M; Benedicenti F; Antonioli G; Assanelli A; Cicalese MP; Del Carro U; Sora MG; Martino S; Quattrini A; Montini E; Di Serio C; Ciceri F; Roncarolo MG; Aiuti A; Naldini L; Biffi A
Lancet; 2016 Jul; 388(10043):476-87. PubMed ID: 27289174
[TBL] [Abstract][Full Text] [Related]
18. Microglia damage precedes major myelin breakdown in X-linked adrenoleukodystrophy and metachromatic leukodystrophy.
Bergner CG; van der Meer F; Winkler A; Wrzos C; Türkmen M; Valizada E; Fitzner D; Hametner S; Hartmann C; Pfeifenbring S; Stoltenburg-Didinger G; Brück W; Nessler S; Stadelmann C
Glia; 2019 Jun; 67(6):1196-1209. PubMed ID: 30980503
[TBL] [Abstract][Full Text] [Related]
19. Treatment of X-linked childhood cerebral adrenoleukodystrophy by the use of an allogeneic stem cell transplantation with reduced intensity conditioning regimen.
Resnick IB; Abdul Hai A; Shapira MY; Bitan M; Hershkovitz E; Schwartz A; Ben-Harush M; Or R; Slavin S; Kapelushnik J
Clin Transplant; 2005 Dec; 19(6):840-7. PubMed ID: 16313334
[TBL] [Abstract][Full Text] [Related]
20. Differential outcomes for frontal versus posterior demyelination in childhood cerebral adrenoleukodystrophy.
Gupta AO; Nascene DR; Shanley R; Kenney-Jung DL; Eisengart JB; Lund TC; Orchard PJ; Pierpont EI
J Inherit Metab Dis; 2021 Nov; 44(6):1434-1440. PubMed ID: 34499753
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
