Translarna
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Active Ingredient: ataluren
Product Overview
About Translarna
Translarna is a medicine that has received a conditional marketing authorisation.
What it is used for
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.
How it is used
This information is not available in the provided facts.
How it works
This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required.
Active substance: ataluren
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