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BIIB065, ISIS-DMPK-2.5Rx (formerly IONIS-DMPKRx, ISIS-DMPKRx)

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Company Ionis Pharmaceuticals Inc.
DescriptionAntisense therapeutic targeting dystrophia myotonica-protein kinase (DMPK)
Molecular Target Dystrophia myotonica-protein kinase (DMPK)
Mechanism of ActionGene silencing
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentPhase I/II
Standard IndicationMyotonic dystrophy type 1 (DM1)
Indication DetailsTreat myotonic dystrophy type 1 (DM1)
Regulatory Designation U.S. - Orphan Drug (Treat myotonic dystrophy type 1 (DM1))
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