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 399:372-383, Fumagalli, F.,et al, 2022
Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
NEJM 385:1929-1940, Gentner, B.,et al, 2021
Autologous Hematopoietic Stem Cell Transplantation for Stiff-Person Spectrum Disorder
Neurol 96:e811-e830, Burt, R.K.,et al, 2021
Long-Term Outcomes after Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis
JAMA Neurol 74:459-469,392, Muraro, P.A.,et al, 2017
Myasthenia Gravis Treated with Autologous Hematopoietic Stem Cell Transplantation
JAMA Neurol 73:652-658,624, Bryant, A.,et al, 2016
Stem Cell Transplantation for Ischemic Stroke
Stroke 42:e33, Boncoraglio,G.B.,et al, 2011