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Phase 3
Development Phase
0
Upcoming Catalysts
0
Historical Events
1
Regulatory Approvals
No Catalysts Found
No catalyst events have been recorded for this drug yet.
Competitive Landscape
10 competitors in Duchenne Muscular Dystrophy
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Patent & Exclusivity
No patent data available. Pre-approval drugs may not have Orange Book listings.
Regulatory Approvals
EMA7/31/2014
Translarna
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.
Conditional
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Mechanism of action & safety profile
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