PTC124 Size:Solid 2 mg InChi: InChI=1S/C10H14N8
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PTC124 Size:Solid 2 mg InChi: InChI=1S/C10H14N8Ataluren, also known as PTC124, is an investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. Ataluren is currently being investigated for use in patients with nonsense mutation Duchenne Becker muscular dystrophy (nmDBMD) and cystic fibrosis
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