230 related articles for article (PubMed ID: 23348427)
1. 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]
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. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease.
Escolar ML; Poe MD; Provenzale JM; Richards KC; Allison J; Wood S; Wenger DA; Pietryga D; Wall D; Champagne M; Morse R; Krivit W; Kurtzberg J
N Engl J Med; 2005 May; 352(20):2069-81. PubMed ID: 15901860
[TBL] [Abstract][Full Text] [Related]
4. 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]
5. Outcomes of unrelated umbilical cord blood transplantation for X-linked adrenoleukodystrophy.
Beam D; Poe MD; Provenzale JM; Szabolcs P; Martin PL; Prasad V; Parikh S; Driscoll T; Mukundan S; Kurtzberg J; Escolar ML
Biol Blood Marrow Transplant; 2007 Jun; 13(6):665-74. PubMed ID: 17531776
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
Fumagalli F; Zambon AA; Rancoita PMV; Baldoli C; Canale S; Spiga I; Medaglini S; Penati R; Facchini M; Ciotti F; Sarzana M; Lorioli L; Cesani M; Natali Sora MG; Del Carro U; Cugnata F; Antonioli G; Recupero S; Calbi V; Di Serio C; Aiuti A; Biffi A; Sessa M
J Inherit Metab Dis; 2021 Sep; 44(5):1151-1164. PubMed ID: 33855715
[TBL] [Abstract][Full Text] [Related]
8. Outcome of Early Juvenile Onset Metachromatic Leukodystrophy After Unrelated Cord Blood Transplantation: A Case Series and Review of the Literature.
Chen X; Gill D; Shaw P; Ouvrier R; Troedson C
J Child Neurol; 2016 Mar; 31(3):338-44. PubMed ID: 26187619
[TBL] [Abstract][Full Text] [Related]
9. Insights into the natural history of metachromatic leukodystrophy from interviews with caregivers.
Harrington M; Whalley D; Twiss J; Rushton R; Martin S; Huynh L; Yang H
Orphanet J Rare Dis; 2019 Apr; 14(1):89. PubMed ID: 31036045
[TBL] [Abstract][Full Text] [Related]
10. Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Fumagalli F; Calbi V; Natali Sora MG; Sessa M; Baldoli C; Rancoita PMV; Ciotti F; Sarzana M; Fraschini M; Zambon AA; Acquati S; Redaelli D; Attanasio V; Miglietta S; De Mattia F; Barzaghi F; Ferrua F; Migliavacca M; Tucci F; Gallo V; Del Carro U; Canale S; Spiga I; Lorioli L; Recupero S; Fratini ES; Morena F; Silvani P; Calvi MR; Facchini M; Locatelli S; Corti A; Zancan S; Antonioli G; Farinelli G; Gabaldo M; Garcia-Segovia J; Schwab LC; Downey GF; Filippi M; Cicalese MP; Martino S; Di Serio C; Ciceri F; Bernardo ME; Naldini L; Biffi A; Aiuti A
Lancet; 2022 Jan; 399(10322):372-383. PubMed ID: 35065785
[TBL] [Abstract][Full Text] [Related]
11. Haematopoietic stem cell transplantation does not retard disease progression in the psycho-cognitive variant of late-onset metachromatic leukodystrophy.
Smith NJ; Marcus RE; Sahakian BJ; Kapur N; Cox TM
J Inherit Metab Dis; 2010 Dec; 33 Suppl 3():S471-5. PubMed ID: 21080229
[TBL] [Abstract][Full Text] [Related]
12. Umbilical cord blood transplantation for juvenile metachromatic leukodystrophy.
Pierson TM; Bonnemann CG; Finkel RS; Bunin N; Tennekoon GI
Ann Neurol; 2008 Nov; 64(5):583-7. PubMed ID: 19067349
[TBL] [Abstract][Full Text] [Related]
13. Multifocal slowing of nerve conduction in metachromatic leukodystrophy.
Cameron CL; Kang PB; Burns TM; Darras BT; Jones HR
Muscle Nerve; 2004 Apr; 29(4):531-6. PubMed ID: 15052618
[TBL] [Abstract][Full Text] [Related]
14. Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: a 5-year follow-up in three affected siblings.
Cable C; Finkel RS; Lehky TJ; Biassou NM; Wiggs EA; Bunin N; Pierson TM
Mol Genet Metab; 2011 Feb; 102(2):207-9. PubMed ID: 21035368
[TBL] [Abstract][Full Text] [Related]
15. Clinical and biochemical study of 29 Brazilian patients with metachromatic leukodystrophy.
Artigalás O; Lagranha VL; Saraiva-Pereira ML; Burin MG; Lourenço CM; van der Linden H; Santos ML; Rosemberg S; Steiner CE; Kok F; de Souza CF; Jardim LB; Giugliani R; Schwartz IV
J Inherit Metab Dis; 2010 Dec; 33 Suppl 3():S257-62. PubMed ID: 20596894
[TBL] [Abstract][Full Text] [Related]
16. Neurophysiology and MRI in late-infantile metachromatic leukodystrophy.
Zafeiriou DI; Kontopoulos EE; Michelakakis HM; Anastasiou AL; Gombakis NP
Pediatr Neurol; 1999 Nov; 21(5):843-6. PubMed ID: 10593679
[TBL] [Abstract][Full Text] [Related]
17. Metachromatic leukodystrophy: natural course of cerebral MRI changes in relation to clinical course.
Groeschel S; Kehrer C; Engel C; I Dali C; Bley A; Steinfeld R; Grodd W; Krägeloh-Mann I
J Inherit Metab Dis; 2011 Oct; 34(5):1095-102. PubMed ID: 21698385
[TBL] [Abstract][Full Text] [Related]
18. Language and cognition in children with metachromatic leukodystrophy: onset and natural course in a nationwide cohort.
Kehrer C; Groeschel S; Kustermann-Kuhn B; Bürger F; Köhler W; Kohlschütter A; Bley A; Steinfeld R; Gieselmann V; Krägeloh-Mann I;
Orphanet J Rare Dis; 2014 Feb; 9():18. PubMed ID: 24499656
[TBL] [Abstract][Full Text] [Related]
19. Presymptomatic late-infantile metachromatic leukodystrophy treated with bone marrow transplantation.
Pridjian G; Humbert J; Willis J; Shapira E
J Pediatr; 1994 Nov; 125(5 Pt 1):755-8. PubMed ID: 7965430
[TBL] [Abstract][Full Text] [Related]
20.
; ; . PubMed ID:
[No Abstract] [Full Text] [Related]
[Next] [New Search]
