Detalhe da pesquisa
1.
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.
Lancet
; 399(10322): 372-383, 2022 01 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-35065785
2.
Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
J Inherit Metab Dis
; 44(5): 1151-1164, 2021 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-33855715
3.
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.
Lancet
; 388(10043): 476-87, 2016 Jul 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-27289174
4.
Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy.
Bone Marrow Transplant
; 54(12): 1995-2003, 2019 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-31150018
5.
Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.
Bone Marrow Transplant
; 53(7): 913-917, 2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29379168