TY  - GEN
AU  - Mercati,Oriane
AU  - Pichard,Samia
AU  - Ouachée,Marie
AU  - Froissart,Roseline
AU  - Fenneteau,Odile
AU  - Roche,Bastien
AU  - Elmaleh-Bergès,Monique
AU  - Bertrand,Yves
AU  - Ogier de Baulny,Hélène
AU  - Vanier,Marie T
AU  - Schiff,Manuel
TI  - Limited benefits of presymptomatic cord blood transplantation in neurovisceral acid sphingomyelinase deficiency (ASMD) intermediate type
SN  - 1532-2130
PY  - 2018///0509
KW  - Child
KW  - Cord Blood Stem Cell Transplantation
KW  - methods
KW  - Female
KW  - Homozygote
KW  - Humans
KW  - Male
KW  - Mutation
KW  - Niemann-Pick Diseases
KW  - therapy
KW  - Pedigree
KW  - Siblings
KW  - Sphingomyelin Phosphodiesterase
KW  - deficiency
KW  - Treatment Outcome
N1  - Publication Type: Case Reports; Journal Article
UR  - https://doi.org/10.1016/j.ejpn.2017.07.015
ER  -