A 23-Year-Old Woman Presenting with Cognitive Impairment and Gait Disturbance
Neurol 99:997-1003, Chaity,D.K.,et al, 2022
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
Long-Term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Juvenile Metachromatic Leukodystrophy Compared with Nontransplanted Control Patients
JAMA Neurol 73:1133-1140, Groeschel, S.,et al, 2016
Late Juvenile Metachromatic Leukodystrophy Treated with Bone Marrow Transplantation:A 4-Year Follow-up Study
Neurol 46:254-256, Navarro,C.,et al, 1996
Longitudinal Neurophysiologic Studies in Pt with Metachromatic Leukodystrophy Following Bone Marrow Trnspl
Arch Neurol 49:1088-1092, Dhuna,A.,et al, 1992
Cholelithiasis and Wilson Disease
J Pediatr 2:210-213, Rosenfield,N.,et al, 1978