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
- -
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
- Conditions
- Interventions
- First Posted Date
- 2009-02-19
- Last Posted Date
- 2020-07-15
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 173
- Registration Number
- NCT00847379
- Locations
- 🇪🇸
Hospital Universitario La Fe, Valencia, Spain
🇦🇺The Children's Hospital at Westmead, Westmead, Australia
🇺🇸Child Neurology Center of NW Florida, Gulf Breeze, Florida, United States
Study of Ataluren (PTC124™) in Cystic Fibrosis
- First Posted Date
- 2008-12-05
- Last Posted Date
- 2020-05-14
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 238
- Registration Number
- NCT00803205
- Locations
- 🇺🇸
Miller Children's Hospital Long Beach, Long Beach, California, United States
🇺🇸University of Iowa, Iowa City, Iowa, United States
🇺🇸University of Texas, Tyler, Texas, United States
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
- First Posted Date
- 2008-09-25
- Last Posted Date
- 2020-10-29
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 36
- Registration Number
- NCT00759876
- Locations
- 🇺🇸
University of Utah, Salt Lake City, Utah, United States
🇺🇸Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
🇺🇸Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
- Conditions
- Interventions
- First Posted Date
- 2008-01-14
- Last Posted Date
- 2020-04-07
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 174
- Registration Number
- NCT00592553
- Locations
- 🇺🇸
University of Minnesota, Minneapolis, Minnesota, United States
🇦🇺Royal Children's Hospital, Parkville, Victoria, Australia
🇺🇸Children's Hospital of Boston/Harvard Medical School, Boston, Massachusetts, United States
A Study of Ataluren in Pediatric Participants With Cystic Fibrosis
- First Posted Date
- 2007-04-10
- Last Posted Date
- 2020-03-06
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 30
- Registration Number
- NCT00458341
- Locations
- 🇧🇪
Reine Fabiola Hospital, Brussels, Belgium
🇫🇷Hopital Necker Enfants Malades, Paris, France
🇧🇪UZ Gasthuisberg Leuven, Leuven, Belgium
Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis
- First Posted Date
- 2005-10-12
- Last Posted Date
- 2020-06-11
- Lead Sponsor
- PTC Therapeutics
- Target Recruit Count
- 24
- Registration Number
- NCT00237380
- Locations
- 🇮🇱
Hadassah University Hospital - Mount Scopus, Jerusalem, Israel
Drug Development Updates
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