Evidence map›Paper›PMID 38930117›Full record

ArticleJournal of clinical medicine2024

Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry.

Deborah Elstein, Nadia Belmatoug, Bruno Bembi, Patrick Deegan, Diego Fernandez-Sasso, Pilar Giraldo, Özlem Göker-Alpan, Derralynn Hughes, Heather Lau, Elena Lukina and 8 more

Abstract read
In one paragraph

Article in Journal of clinical medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing 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

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

  1. Pooled it
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Obstacles to Early Diagnosis of Gaucher Disease.Therapeutics and clinical risk management · 2025
    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

18 authors.

Deborah ElsteinTakeda Pharmaceuticals International AG, 8152 Zurich, Switzerland.ORCID 0000-0001-7092-0126
Nadia BelmatougAssistance-Publique Hôpitaux de Paris Nord, Université Paris Cité, 92110 Clichy, France.ORCID 0000-0002-7917-741X
Bruno BembiCentre for Lysosomal Diseases, Academic Medical Centre Hospital of Udine, 33100 Udine, Italy.
Patrick DeeganDepartment of Medicine, Addenbrookes Hospital, University of Cambridge, Cambridge CB2 0QQ, UK.ORCID 0000-0001-9427-9900
Diego Fernandez-SassoInstituto William Osler, Buenos Aires C1425 BRI, Argentina.
Pilar GiraldoCIBER de Enfermedades Raras, IIS Aragon, 50009 Zaragoza, Spain.ORCID 0000-0002-8791-1901
Özlem Göker-AlpanLysosomal Disorders Unit and Center for Clinical Trials, O and O Alpan LLC, Fairfax, VA 22030, USA.ORCID 0000-0003-4758-6793
Derralynn HughesLysosomal Storage Disorders Unit, Department of Haematology, Royal Free Hospital, UCL Medical School, London NW3 2QG, UK.
Heather LauLangone Medical Cessnter, New York University, New York, NY 10016, USA.ORCID 0000-0003-1690-9457
Elena LukinaDepartment of Orphan Diseases, National Medical Research Center for Hematology, 125167 Moscow, Russia.
Shoshana Revel-VilkGaucher Unit, The Eisenberg R&D Authority, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.ORCID 0000-0001-9151-0337
Ida Vanessa D SchwartzGenetics Department, Federal University of Rio Grande do Sul (UFRGS), Medical Genetics Service-Clinic Hospital of Porto Alegre, Porto Alegre 90010-150, Brazil.
Majdolen IstaitiGaucher Unit, The Eisenberg R&D Authority, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.ORCID 0000-0001-7598-2828
Jaco BothaTakeda Pharmaceuticals International AG, 8152 Zurich, Switzerland.
Noga GadirTakeda Pharmaceuticals International AG, 8152 Zurich, Switzerland.
Jörn SchenkTakeda Pharmaceuticals International AG, 8152 Zurich, Switzerland.
Ari ZimranGaucher Unit, The Eisenberg R&D Authority, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.ORCID 0000-0003-0077-8608
GOS Study Group

Funding

Takeda (Switzerland) Not applicable
6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

enzyme replacement therapyERTGaucher diseaseGOSregistryvelaglucerase alfa

Identifiers

PMID38930117
PMCPMC11204885

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.