Ataluren
- Generic Name
- Ataluren
- Brand Names
- Translarna
- Drug Type
- Small Molecule
- Chemical Formula
- C15H9FN2O3
- CAS Number
- 775304-57-9
- Unique Ingredient Identifier
- K16AME9I3V
- Background
Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.
- Indication
Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
- Associated Therapies
- -
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