PTC Therapeutics today announced that Translarna™ (ataluren) has been granted marketing approval in the Russian Federation for nonsense mutation Duchenne muscular dystrophy (nmDMD). It is estimated that 13% of DMD patients have a nonsense mutation. In countries where it is approved, Translarna is currently the only medicine that targets the underlying cause of nmDMD.
Translarna marketing approval has been granted by the Ministry of Health of the Russian Federation for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in patients 2 years and older. Primarily affecting males, DMD is a rare, irreversible, and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-twenties due to heart and respiratory failure.2 It is caused by the lack of functional dystrophin protein.2