Evidence map›Paper›PMID 39282076›Full record

ArticleMolecular therapy. Methods & clinical development2024

Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis.

Karen J Quadrini, Catherine Vrentas, Christian Duke, Chris Wilson, Christian J Hinderer, David A Weinstein, Samiah A Al-Zaidy, Susan E Browne, James M Wilson, Yan G Ni

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

10 authors.

Karen J QuadriniPassage Bio, Inc., Philadelphia, PA, USA.
Catherine VrentasPPD, part of Thermo Fisher Scientific, Wilmington, NC, USA.
Christian DukePPD, part of Thermo Fisher Scientific, Wilmington, NC, USA.
Chris WilsonPPD, part of Thermo Fisher Scientific, Wilmington, NC, USA.
Christian J HindererGene Therapy Program, Department of Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
David A WeinsteinPassage Bio, Inc., Philadelphia, PA, USA.
Samiah A Al-ZaidyPassage Bio, Inc., Philadelphia, PA, USA.
Susan E BrownePassage Bio, Inc., Philadelphia, PA, USA.
James M WilsonGene Therapy Program, Department of Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Yan G NiPassage Bio, Inc., Philadelphia, PA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

GM1-gangliosidosis (GM1) is a lysosomal storage disorder caused by mutations in the galactosidase beta 1 gene (

Indexed as

biomarkerenzyme activityfluorometric assayGM1-gangliosidosislysosomal storage diseaseβ-galactosidase-1 deficiency

Identifiers

PMID39282076
PMCPMC11401230

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.