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ataluren (Translarna): Dosing and Uses

 

Classes: Neurologics, Other

Medically reviewed by Min Clinic Staff | Updated: January 2026

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