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drisapersen (2402968) (GSK2402968, PRO051)

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Collapse Summary General Information
Company Prosensa Holding N.V.
DescriptionAntisense oligoribonucleotide that induces exon 51 skipping on the dystrophin gene
Molecular Target
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentRegistration
Standard IndicationMuscular dystrophy
Indication DetailsTreat Duchenne muscular dystrophy (DMD)
Regulatory Designation

U.S. - Breakthrough Therapy (Treat Duchenne muscular dystrophy (DMD));
U.S. - Orphan Drug (Treat Duchenne muscular dystrophy (DMD));
EU - Orphan Drug (Treat Duchenne muscular dystrophy (DMD));
Japan - Orphan Drug (Treat Duchenne muscular dystrophy (DMD));
Australia - Orphan Drug (Treat Duchenne muscular dystrophy (DMD))

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