Patterson, Marc C <br/><br/>
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study.  [electronic resource] 

 -  Orphanet journal of rare diseases  May 2015 
 - 65 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1750-1172 
<br/><br/><label>Standard No.: </label>10.1186/s13023-015-0284-z  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>1-Deoxynojirimycin--analogs & derivatives<br/>Adolescent<br/>Adult<br/>Child<br/>Child, Preschool<br/>Cohort Studies<br/>Disease Progression<br/>Enzyme Inhibitors--therapeutic use<br/>Female<br/>Humans<br/>Male<br/>Niemann-Pick Disease, Type C--drug therapy<br/>Prospective Studies<br/>Treatment Outcome<br/>Young Adult