Ataluren
CAS 775304-57-9 2 FDA-DMF suppliersSmall Molecule
Overview
Ataluren is a small-molecule investigational drug designed to facilitate ribosomal readthrough of premature stop codons in mRNA. It is being developed for the treatment of genetic disorders caused by nonsense mutations, such as Duchenne Muscular Dystrophy.
Therapeutic areas
Muscular DystrophyGenetic Disorders
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