Evidence map›Paper›PMID 37373710›Full record

ArticleJournal of clinical medicine2023

Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS).

Derralynn A Hughes, Patrick Deegan, Pilar Giraldo, Özlem Göker-Alpan, Heather Lau, Elena Lukina, Shoshana Revel-Vilk, Maurizio Scarpa, Jaco Botha, Noga Gadir and 2 more

Open access · goldAbstract read
In one paragraph

Article in Journal of clinical medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
0.2field-weighted citation impact, top 49% of its field
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

0 citing papers in PubMed, 1 citations in OpenAlex.

No citing paper in PubMed yet.

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 at 8 institutions in 6 countries.

Derralynn A HughesLSD Unit, Royal Free London NHS Foundation Trust, University College London, London NW3 2QG, UK.
Patrick DeeganAddenbrookes Hospital, Cambridge CB2 0QQ, UK.ORCID 0000-0001-9427-9900
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&O Alpan LLC, Fairfax, VA 22030, USA.ORCID 0000-0003-4758-6793
Heather LauDepartment of Neurology, New York University School of Medicine, New York, NY 10016, USA.
Elena LukinaDepartment of Orphan Diseases, National Research Center for Hematology, 125167 Moscow, Russia.
Shoshana Revel-VilkGaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.ORCID 0000-0001-9151-0337
Maurizio ScarpaCentre for Rare Diseases, Academic Medical Centre Hospital of Udine, 33100 Udine, Italy.
Jaco BothaTakeda Pharmaceuticals International AG, 8152 Zurich, Switzerland.
Noga GadirTakeda Pharmaceuticals International AG, 8152 Zurich, Switzerland.
Ari ZimranGaucher Unit, Shaare Zedek Medical Center, Jerusalem 9103102, Israel.ORCID 0000-0003-0077-8608
GOS Steering Committee
Hebrew University of Jerusalem · ILTakeda (Switzerland) · CHAddenbrooke's Hospital · GBCentro de Investigación Biomédica en Red · ESLysosomal and Rare Disorders Research and Treatment Center · USNational Medical Research Center for Hematology · RUNew York University · USRoyal Free London NHS Foundation Trust · GB

Funding

The Gaucher Outcome Survey (GOS) is sponsored by Takeda Pharmaceutical Company Limited. R and D contracts with each site
6 · The paper itself

Abstract

Thank you for allowing us the opportunity to respond to the commentary from Mistry and colleagues (Comment: The two substrate reduction therapies for type 1 Gaucher disease are not equivalent) [...].

Identifiers

PMID37373710
PMCPMC10299381
OpenAlexW4380630600

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.