Effects of IONIS-HTTRx in Patients with Early Huntington’s Disease, Results of the First HTT-Lowering Drug Trial (CT.002) - Concepedia

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Effects of IONIS-HTTRx in Patients with Early Huntington’s Disease, Results of the First HTT-Lowering Drug Trial (CT.002)

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References

2018

Year

Sarah J. Tabrizi, Blair R. Leavitt, Holly Kordasiewicz, Christian Czech, Eric E. Swayze, Daniel A. Norris, Tiffany Baumann, Irene Gerlach, Scott Schobel, Anne Smith,

Abstract

To characterize safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of IONIS-HTTRx, an antisense oligonucleotide (ASO) designed to target huntingtin (HTT) mRNA, in patients with early manifest Huntington’s disease (HD).