Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: a 5-year follow-up in three affected siblings.
Cable
Casey
C
Neurogenetics Branch, National Institute of Neurological Disorders and Stroke/NIH, Bethesda, MD 20892, USA.
Finkel
Richard S
RS
Lehky
Tanya J
TJ
Biassou
Nadia M
NM
Wiggs
Edythe A
EA
Bunin
Nancy
N
Pierson
Tyler Mark
TM
D000293:Adolescent; D001921:Brain / Q000000981:diagnostic imaging / Q000201:enzymology / Q000473:pathology; D002553:Cerebroside-Sulfatase / Q000235:genetics / Q000378:metabolism; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007966:Leukodystrophy, Metachromatic / Q000175:diagnosis / Q000503:physiopathology / Q000628:therapy*; D008279:Magnetic Resonance Imaging; D008297:Male; D011877:Radionuclide Imaging; D035781:Siblings; D016896:Treatment Outcome
D002553:Cerebroside-Sulfatase