Evidence map›Paper›PMID 36878704›Full record

ArticleNeurology2023

New Therapeutic Approaches to Inherited Metabolic Pediatric Epilepsies.

Phillip L Pearl, Itay Tokatly Latzer, Henry H C Lee, Alexander Rotenberg

Abstract read
In one paragraph

Article in Neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
–field-weighted citation impact
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

8 citing papers in PubMed.

  1. Review
  2. Review
  3. Review
  4. Article
  5. Review
  6. The neurology of creativity: 2023 Hower lecture.Annals of the Child Neurology Society · 2024
    Review
  7. Review
  8. Review
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

4 authors.

Phillip L PearlFrom the Department of Neurology (P.L.P., I.T.L., H.H.C.L., A.R.), Boston Children's Hospital, Harvard Medical School, Boston, MA. phillip.pearl@childrens.harvard.edu.ORCID 0000-0002-6373-1068
Itay Tokatly LatzerFrom the Department of Neurology (P.L.P., I.T.L., H.H.C.L., A.R.), Boston Children's Hospital, Harvard Medical School, Boston, MA.
Henry H C LeeFrom the Department of Neurology (P.L.P., I.T.L., H.H.C.L., A.R.), Boston Children's Hospital, Harvard Medical School, Boston, MA.
Alexander RotenbergFrom the Department of Neurology (P.L.P., I.T.L., H.H.C.L., A.R.), Boston Children's Hospital, Harvard Medical School, Boston, MA.

Funding

Genetic Analysis and Manipulation Core (GAEC)P50HD105351 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI Hisashi Umemori · 2021 to 2026
$9.4M
Developing an inducible mouse model for gene replacement therapy in Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD)R21NS121858 · NINDS · BOSTON CHILDREN'S HOSPITAL · PI ROTENBERG, ALEXANDER · 2022 to 2023
$487k
NICHD NIH HHS P50 HD105351NINDS NIH HHS R21 NS121858
6 · The paper itself

Abstract

Treatment options for inherited metabolic epilepsies are rapidly expanding with advances in molecular biology and the genomic revolution. Traditional dietary and nutrient modification and inhibitors or enhancers of protein and enzyme function, the mainstays of therapy, are undergoing continuous revisions to increase biological activity and reduce toxicity. Enzyme replacement and gene replacement and editing hold promise for genetically targeted treatment and cures. Molecular, imaging, and neurophysiologic biomarkers are emerging as key indicators of disease pathophysiology, severity, and response to therapy.

Indexed as

EpilepsyChildHumans

Identifiers

PMID36878704
PMCPMC10382274

What OpenQuestion holds

Textmetadata
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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.