Search results for "ORPHAN DRUG|Designated/Withdrawn|Treatment of aniridia" in comments (approximate match)
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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...