Last reviewed · How we verify
PTC124
At a glance
| Generic name | PTC124 |
|---|---|
| Sponsor | PTC Therapeutics |
| Modality | Small molecule |
| Phase | Phase 2 |
Approved indications
Common side effects
Key clinical trials
- Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy (PHASE3)
- Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) (PHASE3)
- Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
- A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (PHASE2)
- Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome (PHASE2)
- Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorectal Carcinoma or Metastatic dMMR Endometrial Carcinoma: the ATAPEMBRO Study (PHASE1, PHASE2)
- Study of Ataluren in Participants With Nonsense Mutation Aniridia (PHASE2)
- A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- PTC124 CI brief — competitive landscape report
- PTC124 updates RSS · CI watch RSS
- PTC Therapeutics portfolio CI