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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. Fumagalli F, et al. Among authors: schwab lc. Lancet. 2022 Jan 22;399(10322):372-383. doi: 10.1016/S0140-6736(21)02017-1. Lancet. 2022. PMID: 35065785 Free PMC article. Clinical Trial.
Hippocampal dysfunction defines disease onset in Huntington's disease.
Begeti F, Schwab LC, Mason SL, Barker RA. Begeti F, et al. Among authors: schwab lc. J Neurol Neurosurg Psychiatry. 2016 Sep;87(9):975-81. doi: 10.1136/jnnp-2015-312413. Epub 2016 Feb 1. J Neurol Neurosurg Psychiatry. 2016. PMID: 26833174 Clinical Trial.
Dopamine and Huntington's disease.
Schwab LC, Garas SN, Drouin-Ouellet J, Mason SL, Stott SR, Barker RA. Schwab LC, et al. Expert Rev Neurother. 2015 Apr;15(4):445-58. doi: 10.1586/14737175.2015.1025383. Epub 2015 Mar 16. Expert Rev Neurother. 2015. PMID: 25773746 Review.
Erratum. Correction to: Dopamine and Huntington's disease.
Schwab LC, Garas SN, Drouin-Ouellet J, Mason SL, Stott SR, Barker RA. Schwab LC, et al. Expert Rev Neurother. 2015;15(11):1367. doi: 10.1586/14737175.2015.1088179. Epub 2015 Nov 2. Expert Rev Neurother. 2015. PMID: 26523752 No abstract available.