Ataluren is a small compound that enables ribosome read-through of mRNA containing such a premature stop codon, resulting in production of a full-length protein.



Duchenne Muscular Dystrophy resulting from a non sense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older.


Commercial Presentation

Translarna™ is marketed as sachets of 100 mg, 125 mg & 250 mg packaged in a box of 30.