Evidence map›Paper›PMID 38792324›Full record

ArticleJournal of clinical medicine2024

Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry.

Patrick Deegan, Heather Lau, Deborah Elstein, Diego Fernandez-Sasso, Pilar Giraldo, Derralynn Hughes, Ari Zimran, Majdolen Istaiti, Noga Gadir, Jaco Botha and 2 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 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

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

  1. Pooled it
  2. Review
  3. Article
  4. 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

12 authors.

Patrick DeeganLysosomal Disorders Unit, Cambridge University Hospitals, Hills Road, Cambridge CB2 0QQ, UK.ORCID 0000-0001-9427-9900
Heather LauLangone Medical Center, New York University, 333 E 33rd St, New York, NY 10016, USA.ORCID 0000-0003-1690-9457
Deborah ElsteinTakeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152 Zurich, Switzerland.ORCID 0000-0001-7092-0126
Diego Fernandez-SassoInstituto William Osler, Paraguay 2935, Buenos Aires C1425 BRI, Argentina.
Pilar GiraldoEn el Centro de Investigación Biomédica en Red (CIBER) de Enfermedades Raras, IIS Aragon, C. de San Juan Bosco 13, 50009 Zaragoza, Spain.ORCID 0000-0002-8791-1901
Derralynn HughesLysosomal Storage Disorders Unit, Department of Haematology, Royal Free Hospital, UCL Medical School, Pond Street, London NW3 2QG, UK.
Ari ZimranGaucher Unit, Shaare Zedek Medical Center, Shmuel (Hans) Beyth St 12, Jerusalem 9103102, Israel.ORCID 0000-0003-0077-8608
Majdolen IstaitiGaucher Unit, Shaare Zedek Medical Center, Shmuel (Hans) Beyth St 12, Jerusalem 9103102, Israel.ORCID 0000-0001-7598-2828
Noga GadirTakeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152 Zurich, Switzerland.
Jaco BothaTakeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152 Zurich, Switzerland.
Shoshana Revel-VilkGaucher Unit, Shaare Zedek Medical Center, Shmuel (Hans) Beyth St 12, Jerusalem 9103102, Israel.ORCID 0000-0001-9151-0337
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 diseaseGOSlong termregistryvelaglucerase alfa

Identifiers

PMID38792324
PMCPMC11122485

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.