Evidence map›Paper›PMID 40725823›Full record

ReviewJournal of clinical medicine2025

Clinical Efficacy and Real-World Effectiveness of Fabry Disease Treatments: A Systematic Literature Review.

Ana Jovanovic, Eve Miller-Hodges, Felicia Castriota, Obaro Evuarherhe, Olulade Ayodele, Derralynn Hughes, Guillem Pintos-Morell, Roberto Giugliani, Sandro Feriozzi, Csaba Siffel

Abstract readReview
In one paragraph

Review in Journal of clinical medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
8citing 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

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

  1. Pooled it
  2. Article
  3. Article
  4. Review
  5. Fabry Disease: A Focus on the Role of Oxidative Stress.Antioxidants (Basel, Switzerland) · 2026
    Review
  6. Review
  7. Article
  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

10 authors.

Ana JovanovicThe Mark Holland Metabolic Unit, Northern Care Alliance NHS Foundation Trust, Salford M6 8HD, UK.
Eve Miller-HodgesCentre for Cardiovascular Science, Queen's Medical Research Institute, University of Edinburgh, Edinburgh EH16 4TJ, UK.ORCID 0000-0002-7687-5781
Felicia CastriotaGlobal Evidence and Outcomes, Data and Quantitative Sciences Institute, Takeda Development Center Americas, Inc., Cambridge, MA 02142, USA.
Obaro EvuarherheOxford PharmaGenesis Ltd., Tubney OX13 5QJ, UK.
Olulade AyodeleGlobal Evidence and Outcomes, Data and Quantitative Sciences Institute, Takeda Development Center Americas, Inc., Cambridge, MA 02142, USA.ORCID 0000-0001-6084-5900
Derralynn HughesLysosomal Storage Disorders Unit, Royal Free London NHS Foundation Trust, University College London, London WC1E 6BT, UK.ORCID 0000-0003-4531-9173
Guillem Pintos-MorellVall d'Hebron Institute of Research, Vall d'Hebron Barcelona Hospital Campus, MPS-Lisosomales Medical Committee, 08035 Barcelona, Spain.ORCID 0000-0002-9347-2386
Roberto GiuglianiDepartment of Genetics, Federal University of Rio Grande do Sul (UFRGS), Medical Genetics Service, Hospital de Clinicas de Porto Alegre (HCPA), Dasa Genomics, and Casa dos Raros, Porto Alegre 90610-261, Brazil.ORCID 0000-0001-9655-3686
Sandro FeriozziNephrology Department, University Campus Bio-Medico, University of Rome, 00128 Rome, Italy.ORCID 0000-0001-8420-8447
Csaba SiffelGlobal Evidence and Outcomes, Data and Quantitative Sciences Institute, Takeda Development Center Americas, Inc., Cambridge, MA 02142, USA.ORCID 0000-0002-6491-6953

Funding

Takeda Development Center Americas, Inc Not applicable
6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

agalsidase alfaagalsidase betaeffectivenessefficacyFabry diseaselysosomal storage diseasemigalastat

Identifiers

PMID40725823
PMCPMC12295217

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.