Ataluren

Chemical formula: C₁₅H₉FN₂O₃ Molecular mass: 284.242 g/mol PubChem compound: 11219835

Therapeutic indications

Ataluren is indicated for:

Duchenne muscular dystrophy

Population group: only infants (40 days - 1 year old) , children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 years old or older)

Ataluren is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older.

The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.

For this indication, competent medicine agencies globally authorize below treatments (click for details):

10 mg/kg body weight in the morning, 10 mg/kg body weight at midday and 20 mg/kg body weight in the evening

Contraindications

Ataluren is contraindicated in the following cases:

Lactation

Pregnancy

Severe renal impairment, end-stage renal disease

at least one of

Renal failure stage 5: Kidney failure (GFR <15 mL/min/1.73 m2 or dialysis)

Intravenous aminoglycosides

Aminoglycoside antibacterials

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