These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.
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]
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. Progressive demyelinating polyneuropathy after hematopoietic cell transplantation in metachromatic leukodystrophy: a case series. Beerepoot S; Boelens JJ; Lindemans C; de Witte MA; Nierkens S; Vrancken AFJE; van der Knaap MS; Bugiani M; Wolf NI J Neurol; 2024 Jul; 271(7):4028-4038. PubMed ID: 38564053 [TBL] [Abstract][Full Text] [Related]