Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease.
Jacobs
J F M
JF
Willemsen
M A A P
MA
Groot-Loonen
J J
JJ
Wevers
R A
RA
Hoogerbrugge
P M
PM
D017485:1-Deoxynojirimycin / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016026:Bone Marrow Transplantation; D002648:Child; D005260:Female; D006801:Humans; D009422:Nervous System Diseases / Q000209:etiology; D013661:Tay-Sachs Disease / Q000150:complications / Q000628:therapy*; D014184:Transplantation, Homologous
D017485:1-Deoxynojirimycin; C059896:miglustat