Ataluren, chemically known as 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid, is a small molecule compound designed to promote "read-through" of premature stop codons in mRNA. Structurally, it contains a benzene ring with a 1,2,4-oxadiazole ring system attached, substituted with a fluorophenyl group. This chemical structure allows ataluren to interact with the ribosomal machinery and induce the incorporation of amino acids at premature stop codons, enabling the continuation of translation and synthesis of full-length, functional proteins.Ataluren is primarily used for the treatment of genetic disorders caused by nonsense mutations, particularly Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF). In DMD, nonsense mutations in the dystrophin gene result in the production of truncated, non-functional dystrophin protein, leading to progressive muscle degeneration and weakness.Ataluren promotes read-through of premature stop codons in dystrophin mRNA, allowing for the synthesis of full-length dystrophin protein, which may improve muscle function and slow disease progression.
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