Translarna™

(ataluren)


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.
 

Indications

 

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.