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. 2021 Jan;36(1):263-264.
doi: 10.1002/mds.28359. Epub 2020 Oct 30.

Hydrocephalus Complicating Intrathecal Antisense Oligonucleotide Therapy for Huntington's Disease

Thomas B Stoker  1 , Katie E R Andresen  1 , Roger A Barker  1   2
Affiliations

Hydrocephalus Complicating Intrathecal Antisense Oligonucleotide Therapy for Huntington's Disease

Thomas B Stoker et al. Mov Disord. 2021 Jan.
No abstract available

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Figures

FIG. 1.
FIG. 1.
Investigation results. Cerebrospinal fluid (CSF) protein (A) and leukocyte count (B). Light and dark gray bars represent treatment with placebo and tominersen, respectively. Arrow indicates clinical deterioration. Axial T2‐weighted magnetic resonance imaging at baseline (C) and after deterioration (D) Lumbar infusion study (E) AMP, pulse amplitude; HR, heart rate; ICP, intracranial pressure; RAP, compensatory reserve index.
See this image and copyright information in PMC

References

    1. Tabrizi SJ, Leavitt BR, Landwehrmeyer GB, et al. Targeting huntingtin expression in patients with Huntington's disease. N Engl J Med 2019;380(24):2307–2316. 10.1056/NEJMoa1900907 - DOI - PubMed
    1. Schobel SA. Preliminary results from a 15‐month open‐label extension study investigating tominersen (RG6042) huntingtin protein antisense oligonucleotide in adults with manifest Huntington's disease. 2020. https://chdifoundation.org/2020-conference/#schobel. Accessed August 12, 2020.
    1. Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile‐onset spinal muscular atrophy. N Engl J Med 2017;377(18):1723–1732. 10.1056/NEJMoa1702752 - DOI - PubMed
    1. Mercuri E, Darras BT, Chiriboga CA, et al. Nusinersen versus sham control in later‐onset spinal muscular atrophy. N Engl J Med 2018;378(7):625–635. 10.1056/NEJMoa1710504 - DOI - PubMed
    1. Spinraza. Product information . www.medicines.org.uk/emc/product/2715. Accessed August 12, 2020.

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