Evidence map›Paper›PMID 42454581›Full record

ArticleJournal of comparative effectiveness research2026

A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa.

Amy Dymond, Will Green, Matthew Crabtree, Alasdair MacCulloch, Neil Johnson, Simon Shohet, Vera Gielen, Sophie Clarke, Patrick Deegan, Jordi Diaz-Manera

Abstract readComparative Study
In one paragraph

Article in Journal of comparative effectiveness research, 2026. 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
–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

0 citing papers in PubMed.

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

10 authors.

Amy DymondYork Health Economics Consortium, York, UK.ORCID 0000-0002-8519-6537
Will GreenYork Health Economics Consortium, York, UK.ORCID 0000-0002-9004-1951
Matthew CrabtreeAmicus Therapeutics UK Ltd, Marlow, UK.
Alasdair MacCullochAmicus Therapeutics UK Ltd, Marlow, UK.
Neil JohnsonAmicus Therapeutics UK Ltd, Marlow, UK.
Simon ShohetFormerly, Amicus Therapeutics UK Ltd, Marlow, UK; Currently, Molecular Economics Ltd, Hitchin, UK.
Vera GielenAmicus Therapeutics UK Ltd, Marlow, UK.
Sophie ClarkeAmicus Therapeutics UK Ltd, Marlow, UK.
Patrick DeeganDepartment of Medicine, Addenbrookes Hospital, University of Cambridge, Cambridge, UK.
Jordi Diaz-ManeraJohn Walton Muscular Dystrophy Research Center, Newcastle University & Translational & Clinical Research Institute, Newcastle Upon Tyne, UK.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

1-Deoxynojirimycinalpha-GlucosidasesGlycogen Storage Disease Type IIAdultDisease ProgressionDrug Therapy, CombinationFemaleHumansMaleMiddle Aged1-Deoxynojirimycinalpha-Glucosidasescipaglucosidase alfaGAA protein, humanmiglustatforced vital capacitypatient-level simulation modelsix-minute walk test

Identifiers

PMID42454581
PMCPMC13435892

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.