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Ionis Pharma (IONS) aims to complete Phase 1/2 study of ISIS-HTTRx (ISIS 443139) in Patients with Early Manifest Huntington's Disease in 2017

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IONS

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Additional Information

Additional Relevant Details First therapy designed to directly target the cause of disease as opposed to treating the symptoms
http://ir.isispharm....
Additional Relevant Details Update on Dec 11 2017: In the Phase 1/2a study, dose-dependent reductions of mHTT were observed in participants treated with IONIS-HTTRx. In addition, the safety and tolerability profile of IONIS-HTTRx observed in the Phase 1/2a study supports continued development. Ionis and Roche plan to present results from this study at medical conferences in the first half of 2018 and plan to submit the study results for publication in a peer-reviewed medical journal. Ionis and Roche have also recently initiated an open-label extension (OLE) study for patients who completed the Phase 1/2a study.
http://ir.ionispharm...
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Related Keywords Phase 1/2 Study, Isis-httrx, Isis 43139, Huntington's Disease