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Translarna
ataluren
Dystrophie musculaire de Duchenne
ℹ️ Expired
✅
Aucune alerte active
Ce médicament ne fait l'objet ni de pénuries en cours, ni de lettres de sécurité, ni de rappels.
À quoi ça sert
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.
⚠️ Sécurité des patients
No
Page officielle de l'EMA
https://www.ema.europa.eu/en/medicines/human/EPAR/translarna