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Showing 1 - 2 of 2 results
Status:
Possibly Marketed Outside US
Source:
NCT03256799: Phase 4 Interventional Completed Cystic Fibrosis
(2017)
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Ataluren (Translarna) is a small-molecule drug approved in Europe for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation. Ataluren interacts with the ribosome enabling it to read through premature nonsense stop signals on mRNA and allowing the cell to produce a full-length, functional protein. Ataluren is also being tested in phase III for cystic fibrosis caused by a nonsense mutation.
