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Cited by 26Genetic Neurodegenerative DiseasesAmyotrophic Lateral Sclerosis ResearchLysosomal Storage Disorders Research

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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)

Tabrizi, Sarah, Leavitt, Blair, Kordasiewicz, Holly, Czech, Christian, Swayze, Eric, Norris, Daniel A., Baumann, Tiffany, Gerlach, Irene, Schobel, Scott, Smith, Anne, Lane, Roger, Bennett, C. Frank·2018·Neurology
DOI 10.1212/wnl.90.15_supplement.ct.002
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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).

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Crossref · 10.1212/wnl.90.15_supplement.ct.002

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Cited by 26

Metadata

Authors
Tabrizi, S., Leavitt, B., Kordasiewicz, H., Czech, C., Swayze, E., Norris, D. A., Baumann, T., Gerlach, I., Schobel, S., Smith, A., Lane, R., & Bennett, C. F.
Affiliations
  • University College London (GB)
  • University of British Columbia (CA)
  • Ionis Pharmaceuticals (United States) (US)
  • F. Hoffmann-La Roche AG (CH)
ORCID
Venue
Neurology
Publisher
Ovid Technologies (Wolters Kluwer Health)
Published
2018-04-10
Volume
90
Issue
15_supplement
ISSN
  • 0028-3878 (ISSN-L)
  • 1526-632X
DOI
10.1212/wnl.90.15_supplement.ct.002
OpenAlex
W2802276893
Cited by
26
References
0
Citations by year
2025: 1 · 2024: 3 · 2023: 1 · 2022: 2 · 2021: 1 · 2020: 2 · 2019: 12 · 2018: 4
FWCI
2.16
Type
journal article
Language
en
Open access
closed
Locations
Neurology (publishedVersion)
Topics
  • Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Genetic Neurodegenerative Diseases
  • Health Sciences › Medicine › Clinical Neurology › Amyotrophic Lateral Sclerosis Research
  • Health Sciences › Medicine › Genetics (clinical) › Lysosomal Storage Disorders Research
Keywords
antisense oligonucleotides, Huntington's disease, safety and tolerability, pharmacokinetics, pharmacodynamics

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