Mercati, Oriane <br/><br/>
Limited benefits of presymptomatic cord blood transplantation in neurovisceral acid sphingomyelinase deficiency (ASMD) intermediate type.  [electronic resource] 

 -  European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society  Nov 2017 
 - 907-911 p.  digital 
<br/><br/> Publication Type: Case Reports; Journal Article 
<br/><br/><label>ISSN: </label>1532-2130 
<br/><br/><label>Standard No.: </label>10.1016/j.ejpn.2017.07.015  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Child<br/>Cord Blood Stem Cell Transplantation--methods<br/>Female<br/>Homozygote<br/>Humans<br/>Male<br/>Mutation<br/>Niemann-Pick Diseases--therapy<br/>Pedigree<br/>Siblings<br/>Sphingomyelin Phosphodiesterase--deficiency<br/>Treatment Outcome