171 related articles for article (PubMed ID: 38326898)
1. Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries.
Schoenmakers DH; Mochel F; Adang LA; Boelens JJ; Calbi V; Eklund EA; Grønborg SW; Fumagalli F; Groeschel S; Lindemans C; Sevin C; Schöls L; Ram D; Zerem A; Graessner H; Wolf NI
Orphanet J Rare Dis; 2024 Feb; 19(1):46. PubMed ID: 38326898
[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. 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]
4. Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management.
Laugwitz L; Schoenmakers DH; Adang LA; Beck-Woedl S; Bergner C; Bernard G; Bley A; Boyer A; Calbi V; Dekker H; Eichler F; Eklund E; Fumagalli F; Gavazzi F; Grønborg SW; van Hasselt P; Langeveld M; Lindemans C; Mochel F; Oberg A; Ram D; Saunier-Vivar E; Schöls L; Scholz M; Sevin C; Zerem A; Wolf NI; Groeschel S
Eur J Paediatr Neurol; 2024 Mar; 49():141-154. PubMed ID: 38554683
[TBL] [Abstract][Full Text] [Related]
5. Hematopoietic Stem Cell Transplantation with Mesenchymal Stromal Cells in Children with Metachromatic Leukodystrophy.
Cabanillas Stanchi KM; Böhringer J; Strölin M; Groeschel S; Lenglinger K; Treuner C; Kehrer C; Laugwitz L; Bevot A; Kaiser N; Schumm M; Lang P; Handgretinger R; Krägeloh-Mann I; Müller I; Döring M
Stem Cells Dev; 2022 Apr; 31(7-8):163-175. PubMed ID: 35323019
[TBL] [Abstract][Full Text] [Related]
6. Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi).
Schoenmakers DH; Beerepoot S; van den Berg S; Adang L; Bley A; Boelens JJ; Fumagalli F; Goettsch WG; Grønborg S; Groeschel S; van Hasselt PM; Hollak CEM; Lindemans C; Mochel F; Mol PGM; Sevin C; Zerem A; Schöls L; Wolf NI
Orphanet J Rare Dis; 2022 Feb; 17(1):48. PubMed ID: 35164810
[TBL] [Abstract][Full Text] [Related]
7. Reduced intensity conditioning haematopoietic stem cell transplantation with mesenchymal stromal cells infusion for the treatment of metachromatic leukodystrophy: a case report.
Meuleman N; Vanhaelen G; Tondreau T; Lewalle P; Kwan J; Bennani J; Martiat P; Lagneaux L; Bron D
Haematologica; 2008 Jan; 93(1):e11-3. PubMed ID: 18166768
[TBL] [Abstract][Full Text] [Related]
8. Allogenic hematopoietic stem cell transplantation in two siblings with adult metachromatic leukodystrophy and a systematic literature review.
Videbæk C; Stokholm J; Sengeløv H; Fjeldborg LU; Larsen VA; Krarup C; Nielsen JE; Grønborg S
JIMD Rep; 2021 Jul; 60(1):96-104. PubMed ID: 34258145
[TBL] [Abstract][Full Text] [Related]
9. 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]
10. Developing therapeutic approaches for metachromatic leukodystrophy.
Patil SA; Maegawa GH
Drug Des Devel Ther; 2013; 7():729-45. PubMed ID: 23966770
[TBL] [Abstract][Full Text] [Related]
11. Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort.
Krägeloh-Mann I; Groeschel S; Kehrer C; Opherk K; Nägele T; Handgretinger R; Müller I
Bone Marrow Transplant; 2013 Mar; 48(3):369-75. PubMed ID: 22941383
[TBL] [Abstract][Full Text] [Related]
12. Understanding caregiver descriptions of initial signs and symptoms to improve diagnosis of metachromatic leukodystrophy.
Eichler F; Sevin C; Barth M; Pang F; Howie K; Walz M; Wilds A; Calcagni C; Chanson C; Campbell L
Orphanet J Rare Dis; 2022 Oct; 17(1):370. PubMed ID: 36195888
[TBL] [Abstract][Full Text] [Related]
13. Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy.
Beschle J; Döring M; Kehrer C; Raabe C; Bayha U; Strölin M; Böhringer J; Bevot A; Kaiser N; Bender B; Grimm A; Lang P; Müller I; Krägeloh-Mann I; Groeschel S
Mol Cell Pediatr; 2020 Sep; 7(1):12. PubMed ID: 32910272
[TBL] [Abstract][Full Text] [Related]
14. Atidarsagene autotemcel for metachromatic leukodystrophy.
Messina M; Gissen P
Drugs Today (Barc); 2023 Feb; 59(2):63-70. PubMed ID: 36811406
[TBL] [Abstract][Full Text] [Related]
15. Metachromatic leukodystrophy: an overview of current and prospective treatments.
Biffi A; Lucchini G; Rovelli A; Sessa M
Bone Marrow Transplant; 2008 Oct; 42 Suppl 2():S2-6. PubMed ID: 18978739
[TBL] [Abstract][Full Text] [Related]
16. Clinical outcome in four children with metachromatic leukodystrophy treated by bone marrow transplantation.
Malm G; Ringdén O; Winiarski J; Gröndahl E; Uyebrant P; Eriksson U; Håkansson H; Skjeldal O; Månsson JE
Bone Marrow Transplant; 1996 Jun; 17(6):1003-8. PubMed ID: 8807106
[TBL] [Abstract][Full Text] [Related]
17. Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network.
Page KM; Stenger EO; Connelly JA; Shyr D; West T; Wood S; Case L; Kester M; Shim S; Hammond L; Hammond M; Webb C; Biffi A; Bambach B; Fatemi A; Kurtzberg J
Biol Blood Marrow Transplant; 2019 Dec; 25(12):e363-e374. PubMed ID: 31499213
[TBL] [Abstract][Full Text] [Related]
18. Gallbladder abnormalities in children with metachromatic leukodystrophy.
Kim J; Sun Z; Ezekian B; Schooler GR; Prasad VK; Kurtzberg J; Rice HE; Tracy ET
J Surg Res; 2017 Feb; 208():187-191. PubMed ID: 27993207
[TBL] [Abstract][Full Text] [Related]
19. 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]
20. The burden of disease in metachromatic leukodystrophy: results of a caregiver survey in the UK and Republic of Ireland.
Thomas S; Morrison A; Morton G; Roberts P; Clark V; Imrie J
Orphanet J Rare Dis; 2024 Feb; 19(1):87. PubMed ID: 38403596
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
