Indications
Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.
Composition
- Active substance
- ataluren
Official product information
The European public assessment report holds the summary of product characteristics and the package leaflet in every EU language.
Prognia shows this product information for reference and learning. It is not treatment advice and can lag behind the official text. Always check the current product information and local protocols before prescribing.
At a glance
- Medicine
- Translarna
- Active substance
- ataluren
- ATC code
- M09AX03
- Therapeutic area
- Muscular Dystrophy, Duchenne
- Marketing authorisation holder
- PTC Therapeutics International Limited