Colaco, Alexandria <br/><br/>
Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease.  [electronic resource] 

 -  Journal of inherited metabolic disease  05 2020 
 - 574-585 p.  digital 
<br/><br/> Publication Type: Journal Article; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1573-2665 
<br/><br/><label>Standard No.: </label>10.1002/jimd.12191  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>1-Deoxynojirimycin--analogs & derivatives<br/>ATP Binding Cassette Transporter 1--genetics<br/>Adult<br/>Humans<br/>Intracellular Signaling Peptides and Proteins--genetics<br/>Middle Aged<br/>Niemann-Pick C1 Protein<br/>Niemann-Pick Disease, Type C--drug therapy<br/>Treatment Outcome