Indication
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.
Medicine details
- Medicine name:
- ataluren (Translarna)
- SMC ID:
- SMC2827
- Pharmaceutical company
- PTC Therapeutics
- BNF chapter
- Musculoskeletal and joint diseases
- Submission type
- Ultra-orphan reassessment
- Publication due date:
- TBC
- SMC meeting date:
- TBC
- Patient group submission deadline:
- TBC