Evidence map›Paper›PMID 30382161›Full record

Trial reportScientific reports2018

A novel therapeutic agent, sodium oxybate, improves dystonic symptoms via reduced network-wide activity.

Kristina Simonyan, Steven J Frucht, Andrew Blitzer, Azadeh Hamzehei Sichani, Anna F Rumbach

Open access · goldAbstract readClinical Trial
In one paragraph

Trial report in Scientific reports, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
1.5field-weighted citation impact, top 18% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

13 citing papers in PubMed, 24 citations in OpenAlex.

  1. Trial
  2. Sodium Oxybate in Alcohol-Responsive Essential Tremor of Voice: An Open-Label Phase II Study.Movement disorders : official journal of the Movement Disorder Society · 2023
    Trial
  3. Article
  4. Review
  5. Review
  6. Review
  7. Article
  8. Review
  9. Review
  10. Alcohol-Responsive Hyperkinetic Movement Disorders-a Mechanistic Hypothesis.Tremor and other hyperkinetic movements (New York, N.Y.) · 2020
    Review
  11. A microstructural neural network biomarker for dystonia diagnosis identified by a DystoniaNet deep learning platform.Proceedings of the National Academy of Sciences of the United States of America · 2020
    Article
  12. Review
  13. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors at 4 institutions in 2 countries.

Kristina SimonyanDepartment of Otolaryngology, Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, MA, United States. Kristina_Simonyan@meei.harvard.edu.ORCID http://orcid.org/0000-0001-7444-0437
Steven J FruchtDepartment of Neurology, New York University, New York, NY, United States.
Andrew BlitzerDepartment of Neurology, Mount Sinai School of Medicine, New York, NY, United States.
Azadeh Hamzehei SichaniDepartment of Otolaryngology, Massachusetts Eye and Ear Infirmary, Harvard Medical School, Boston, MA, United States.
Anna F RumbachSchool of Health and Rehabilitation Sciences, Speech Pathology, The University of Queensland, Brisbane, Queensland, Australia.
Massachusetts Eye and Ear Infirmary · USIcahn School of Medicine at Mount Sinai · USNew York University · USThe University of Queensland · AU

Funding

Voice tremor in spasmodic dysphonia: central mechanisms and treatment responseR01DC012545 · NIDCD · MASSACHUSETTS EYE AND EAR INFIRMARY · PI SIMONYAN, KRISTINA · 2012 to 2021
$5.2M
NIDCD NIH HHS R01 DC012545U.S. Department of Health & Human Services | NIH | National Institute on Deafness and Other Communication Disorders (NIDCD) R01DC012545
6 · The paper itself

Abstract

Oral medications for the treatment of dystonia are not established. Currently, symptoms of focal dystonia are managed with botulinum toxin injections into the affected muscles. However, the injection effects are short-lived and not beneficial for all patients. We recently reported significant clinical improvement of symptoms with novel investigational oral drug, sodium oxybate, in patients with the alcohol-responsive form of laryngeal focal dystonia. Understanding the mechanism of action of this promising oral agent holds a strong potential for the development of a scientific rationale for its use in dystonia. Therefore, to determine the neural markers of sodium oxybate effects, which may underlie dystonic symptom improvement, we examined brain activity during symptomatic speech production before and after drug intake in patients with laryngeal dystonia and compared to healthy subjects. We found that sodium oxybate significantly attenuated hyperfunctional activity of cerebellar, thalamic and primary/secondary sensorimotor cortical regions. Drug-induced symptom improvement was correlated with decreased-to-normal levels of activity in the right cerebellum. These findings suggest that sodium oxybate shows direct modulatory effects on disorder pathophysiology by acting upon abnormal neural activity within the dystonic network.

Indexed as

Botulinum ToxinsBrainDystonic DisordersEthanolFemaleHumansMaleMiddle AgedNerve NetSodium OxybateVoiceBotulinum ToxinsEthanolSodium Oxybate

Identifiers

PMID30382161
PMCPMC6208333
OpenAlexW2898454277

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.