Héron, Bénédicte <br/><br/>
Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C.  [electronic resource] 

 -  Orphanet journal of rare diseases  Jun 2012 
 - 36 p.  digital 
<br/><br/> Publication Type: Clinical Trial; Journal Article 
<br/><br/><label>ISSN: </label>1750-1172 
<br/><br/><label>Standard No.: </label>10.1186/1750-1172-7-36  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>1-Deoxynojirimycin--administration & dosage<br/>Adolescent<br/>Age of Onset<br/>Carrier Proteins--genetics<br/>Child<br/>Child, Preschool<br/>Cohort Studies<br/>Enzyme Inhibitors--administration & dosage<br/>Female<br/>France<br/>Glycoproteins--genetics<br/>Humans<br/>Infant<br/>Intracellular Signaling Peptides and Proteins<br/>Male<br/>Membrane Glycoproteins--genetics<br/>Niemann-Pick C1 Protein<br/>Niemann-Pick Disease, Type C--drug therapy<br/>Prospective Studies<br/>Treatment Outcome<br/>Vesicular Transport Proteins