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Drisapersen
An exon skipping experimental Medicine[1] [2] PRO051/GSK2402968 is considered an orphan drug Duchenne muscular distrophy
Links
Duchenne muscular dystrophy: Still an incurable disease
[https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5010191/ Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study]
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