ataluren

Ataluren is a small molecule drug developed for the treatment of Duchenne muscular dystrophy (DMD) caused by nonsense mutations in the dystrophin gene. It works by enabling ribosomal readthrough of premature stop codons in mRNA, allowing production of full-length, functional dystrophin protein that would otherwise be truncated due to the mutation. Ataluren is orally administered and has received conditional marketing authorization from the European Medicines Agency for ambulatory patients aged 5 years and older with nonsense mutation DMD. It is not approved by the FDA as of June 2025 but remains under review in several countries[1][4][9].

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Other names

3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid