Dosing and uses of Translarna (ataluren)
Duchenne Muscular Dystrophy
Pending FDA approvaL
Aniridia (Orphan)
Orphan designation for treatment of aniridia
Sponsor
- PTC Therapeutics, Inc; 100 Corporate Court; South Plainfield, New Jersey 07080
Pharmacology of Translarna (ataluren)
Mechanism of action
Possibly causes ribosomes to be less sensitive to premature stop codons (ie, ‘read-through’) if mRNA contains a mutation causing premature stop codons or nonsense codons
