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Nonsense Mutation Readthrough Agent
Ataluren for Duchenne Muscular Dystrophy
Recruiting2 awardsPhase 3
London, Ontario
This trial tests the safety and tolerance of ataluren in patients with a specific type of Duchenne Muscular Dystrophy who have been part of previous studies. Ataluren helps the body make important proteins that are usually missing due to genetic errors. The study includes these patients and their siblings who have completed earlier trial phases. Ataluren is the first approved drug for Duchenne muscular dystrophy (DMD) patients with premature stop codon mutations and has been conditionally approved in Europe.
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