Kerem, Eitan <br/><br/>
Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.  [electronic resource] 

 -  Lancet (London, England)  Aug 2008 
 - 719-27 p.  digital 
<br/><br/> Publication Type: Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't 
<br/><br/><label>ISSN: </label>1474-547X 
<br/><br/><label>Standard No.: </label>10.1016/S0140-6736(08)61168-X  doi 
<br/><br/><label>Subjects--Topical Terms: </label><br/>Adolescent<br/>Adult<br/>Chlorides--metabolism<br/>Codon, Nonsense--drug effects<br/>Codon, Terminator--drug effects<br/>Cystic Fibrosis--drug therapy<br/>Cystic Fibrosis Transmembrane Conductance Regulator--drug effects<br/>Female<br/>Humans<br/>Male<br/>Middle Aged<br/>Oxadiazoles--adverse effects<br/>Treatment Outcome