Evidence map›Paper›PMID 37926545›Full record

ArticleInternal medicine (Tokyo, Japan)2024

Intraventricular Cerliponase Alfa Treatment in a Patient with Advanced Neuronal Ceroid Lipofuscinosis Type 2.

Saki Nakashima, Masashi Hamada, Tomohiko Kimura, Shuichi Tanifuji, Akiko Takahashi, Daiki Yashita, Yu Kakimoto, Takashi Matsukawa, Hiroyuki Ishiura, Tatsushi Toda

Abstract readCase Reports
PubMed Full text at PMC Publisher
In one paragraph

Article in Internal medicine (Tokyo, Japan), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

10 authors.

Saki NakashimaDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.
Masashi HamadaDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.
Tomohiko KimuraDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.
Shuichi TanifujiDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.
Akiko TakahashiDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.
Daiki YashitaDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.
Yu KakimotoDepartment of Pediatrics, Graduate School of Medicine, The University of Tokyo, Japan.
Takashi MatsukawaDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.
Hiroyuki IshiuraDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.
Tatsushi TodaDepartment of Neurology, Graduate School of Medicine, The University of Tokyo, Japan.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Neuronal ceroid lipofuscinosis type 2 (CLN2) is an autosomal recessive lysosomal disease caused by decreased activity of the enzyme tripeptidyl peptidase 1 (TPP1) due to pathogenic variants in the TPP1 gene. Cerliponase alfa, a recombinant proenzyme form of TPP1, has shown efficacy in preventing motor and language function decline in early-stage CLN2. However, the safety and effects of this therapy in advanced-stage CLN2 are unclear. We herein report a case of intraventricular cerliponase alfa treatment for over a year in a patient with advanced-stage CLN2. The results suggest the safety and potential efficacy of treatment at an advanced stage of CLN2.

Indexed as

AminopeptidasesDipeptidyl-Peptidases and Tripeptidyl-PeptidasesNeuronal Ceroid-LipofuscinosesSerine ProteasesTripeptidyl-Peptidase 1HumansMaleRecombinant ProteinsTreatment OutcomeYoung AdultAminopeptidasescerliponase alfaDipeptidyl-Peptidases and Tripeptidyl-PeptidasesRecombinant ProteinsSerine ProteasesTPP1 protein, humanTripeptidyl-Peptidase 1cerliponase alfamotor language scoreneuronal ceroid lipofuscinosisseizuretripeptidyl peptidase 1

Identifiers

PMID37926545
PMCPMC11239262

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Registered trials

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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.