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
- -
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
- First Posted Date
- 2010-11-24
- Last Posted Date
- 2025-01-08
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 270
- Registration Number
- NCT01247207
- Locations
- 🇺🇸
Rare Disease Research, LLC, Atlanta, Georgia, United States
🇺🇸Rush Univ Medical Center, Chicago, Illinois, United States
🇺🇸University of Iowa Children's Hospital, Iowa City, Iowa, United States
Drug Development Updates
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