A 23-Year-Old Woman Presenting with Cognitive Impairment and Gait Disturbance
Neurol 99:997-1003, Chaity,D.K.,et al, 2022
Leukodystrophies: Classification, Diagnosis, and Treatment
Neurologist 15:319-328, Costello,D.,et al, 2009
Insights Into the Diagnosis and Treatment of Lysosomal Storage Diseases
Arch Neurol 60:322-328, Wegner,D.A.,et al, 2003
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
Familial Multiple Sclerosis and Other Inherited Disorders of the White Matter
The Neurologist 10:201-215, Kalman,B. &Leist,T.P., 2004
Metachromatic Leukodystrophy:Diffusion MR Imaging Findings
AJNR 23:1424-1426, Sener,R.N., 2002
Long-Term Stabilization After Bone Marrow Transplantation in Juvenile Metachromatic Leukodystrophy
Arch Neurol 55:98-99, Kidd,D.,et al, 1998
Late Juvenile Metachromatic Leukodystrophy Treated with Bone Marrow Transplantation:A 4-Year Follow-up Study
Neurol 46:254-256, Navarro,C.,et al, 1996
Serial MR After Bone Marrow Transplantation in Two Patients with Metachromatic Leukodystrophy
AJNR 15:1929-1932, Stillman,A.E.,et al, 1994
Longitudinal Neurophysiologic Studies in Pt with Metachromatic Leukodystrophy Following Bone Marrow Trnspl
Arch Neurol 49:1088-1092, Dhuna,A.,et al, 1992
Treatment of Late Infantile Metachromatic Leukodystrophy by Bone Marrow Transplantation
NEJM 322:28-32, 541990., Krivit,W.,et al, 1990
Bone-Marrow Transplantation for Neurovisceral Storage Disorders
Editorial, Lancet 2:788-7891986., , 1986
Bone Marrow Transplantation for Metachromatic Leukodystrophy
Lancet 2:471-473, Bayever,E.,et al, 1985
Cholelithiasis and Wilson Disease
J Pediatr 2:210-213, Rosenfield,N.,et al, 1978
Organic Disease Presenting as a Psychiatric Syndrome
Postgrad Med 54:78, , 1973
Neuro CPC of MGH
Metachromatic Leukodystrophy, NEJM 267:1198-12041962., , 1962