Evidence map›Paper›PMID 38448957›Full record

ArticleOrphanet journal of rare diseases2024

Crigler-Najjar syndrome: looking to the future does not make us forget the present.

Fabiola Di Dato, Giuseppe D'Uonno, Raffaele Iorio

Open access · goldAbstract readLetter
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
2.2field-weighted citation impact, top 14% 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

1 citing paper in PubMed, 2 citations in OpenAlex.

  1. 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

3 authors at 2 institutions in 1 country.

Fabiola Di DatoDepartment of Translational Medical Science, Section of Pediatrics, University of Naples Federico II, Via Sergio Pansini 5, 80131, Naples, Italy.
Giuseppe D'UonnoDepartment of Translational Medical Science, Section of Pediatrics, University of Naples Federico II, Via Sergio Pansini 5, 80131, Naples, Italy.
Raffaele IorioDepartment of Translational Medical Science, Section of Pediatrics, University of Naples Federico II, Via Sergio Pansini 5, 80131, Naples, Italy. riorio@unina.it.ORCID 0000-0002-7483-234X
University of Naples Federico II · ITFederico II University Hospital · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Recently, the safety and efficacy of gene therapy were evaluated in patients with Crigler-Najjar syndrome (CNS). Although it is a promising curative option for CNS, many doubts still persist about its long-term efficacy and safety. Furthermore, there is a risk of overlooking several unresolved problems still present in current clinical practice. This letter is a call for action on crucial open issues that remain nowadays an unmet need in the management of CNS patients.

Indexed as

Crigler-Najjar SyndromeEmotionsGenetic TherapyHumansGene therapyLiver transplantationPhenobarbitalPhototherapy

Identifiers

PMID38448957
PMCPMC10918926
OpenAlexW4392559223

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.