Evidence map›Paper›PMID 39273698›Full record

ReviewInternational journal of molecular sciences2024

Establishing Treatment Effectiveness in Fabry Disease: Observation-Based Recommendations for Improvement.

Bram C F Veldman, Daphne H Schoenmakers, Laura van Dussen, Mareen R Datema, Mirjam Langeveld

Abstract readReview
In one paragraph

Review in International journal of molecular sciences, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
–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

5 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Impact of enzyme replacement therapy on hearing sensitivity in Fabry disease: A systematic review.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2026
    Pooled it
  2. Article
  3. Review
  4. Review
  5. 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.

Bram C F VeldmanDepartment of Endocrinology and Metabolism, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.
Daphne H SchoenmakersDepartment of Endocrinology and Metabolism, Platform "Medicine for Society", Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.ORCID 0000-0003-1601-7379
Laura van DussenDepartment of Endocrinology and Metabolism, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.ORCID 0000-0002-6539-531X
Mareen R DatemaDepartment of Endocrinology and Metabolism, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.
Mirjam LangeveldDepartment of Endocrinology and Metabolism, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam UMC Location University of Amsterdam, 1105 AZ Amsterdam, The Netherlands.ORCID 0000-0002-9934-6831

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Fabry disease (FD, OMIM #301500) is caused by pathogenic

Indexed as

alpha-GalactosidaseEnzyme Replacement TherapyFabry Disease1-DeoxynojirimycinFemaleHumansIsoenzymesMaleRecombinant ProteinsTreatment Outcome1-Deoxynojirimycinagalsidase alfaagalsidase betaalpha-GalactosidaseIsoenzymesmigalastatRecombinant Proteinschaperone therapyclinical outcomesenzyme replacement therapy (ERT)Fabry diseasetreatment effectiveness

Identifiers

PMID39273698
PMCPMC11396259

What Socratic 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 Socratic graph.