250mg Translarna Ataluren Sachets

Translarna is a medication indicated for the treatment of Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. It works by enabling ribosomal readthrough of premature stop codons in mRNA, allowing for the production of full-length dystrophin protein. The recommended dose is 10 mg/kg body weight in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening, totaling 40 mg/kg per day. The 250 mg sachet is used in combination with 125 mg and 1000 mg sachets, depending on the patient's weight. The granules should be mixed with at least 30 mL of liquid or 3 tablespoons of semi-solid food before administration. Treatment should be initiated by a specialist experienced in managing DMD. Patients should not take a double dose if a dose is missed and should resume their usual dosing schedule. Co-administration with intravenous aminoglycosides is not recommended due to potential nephrotoxic effects. Patients should maintain adequate hydration during treatment.