TY  - GEN
AU  - Kerem,Eitan
AU  - Hirawat,Samit
AU  - Armoni,Shoshana
AU  - Yaakov,Yasmin
AU  - Shoseyov,David
AU  - Cohen,Michael
AU  - Nissim-Rafinia,Malka
AU  - Blau,Hannah
AU  - Rivlin,Joseph
AU  - Aviram,Micha
AU  - Elfring,Gary L
AU  - Northcutt,Valerie J
AU  - Miller,Langdon L
AU  - Kerem,Batsheva
AU  - Wilschanski,Michael
TI  - Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial
SN  - 1474-547X
PY  - 2008///0909
KW  - Adolescent
KW  - Adult
KW  - Chlorides
KW  - metabolism
KW  - Codon, Nonsense
KW  - drug effects
KW  - Codon, Terminator
KW  - Cystic Fibrosis
KW  - drug therapy
KW  - Cystic Fibrosis Transmembrane Conductance Regulator
KW  - Female
KW  - Humans
KW  - Male
KW  - Middle Aged
KW  - Oxadiazoles
KW  - adverse effects
KW  - Treatment Outcome
N1  - Publication Type: Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't
UR  - https://doi.org/10.1016/S0140-6736(08)61168-X
ER  -