Evidence map›Paper›PMID 39758431›Full record

ArticleJournal of health economics and outcomes research2025

Exploring Quality of Life in Adults Living With Late-onset Pompe Disease: A Combined Quantitative and Qualitative Analysis of Patient Perceptions from Australia, France, Italy, and the Netherlands.

Holly Lumgair, Lisa Bashorum, Alasdair MacCulloch, Elizabeth Minas, George Timmins, Drago Bratkovic, Richard Perry, Medi Stone, Vasileios Blazos, Elisabetta Conti and 1 more

Abstract read
In one paragraph

Article in Journal of health economics and outcomes research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

11 authors.

Holly LumgairAmicus Therapeutics (United Kingdom).
Lisa BashorumAmicus Therapeutics (United Kingdom).
Alasdair MacCullochAmicus Therapeutics (United Kingdom).
Elizabeth MinasAmicus Therapeutics (United Kingdom).
George TimminsConsultant for Amicus Therapeutics, Inc.ORCID https://orcid.org/0000-0001-5302-3941
Drago BratkovicWomen's and Children's Hospital, Adelaide, University of Adelaide.
Richard PerryAdelphi Values PROVE.ORCID https://orcid.org/0000-0001-8790-3170
Medi StoneAdelphi Values PROVE.
Vasileios BlazosAdelphi Values PROVE.
Elisabetta ContiAiGlico, Assago, Italy.
Raymond SaichAustralian Pompe Association, Sydney, Australia.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

PubMed holds no abstract for this paper.

Indexed as

emotional wellbeinglate-onset Pompe diseasepatient perceptionpatient-reported outcomesphysical functioningquality of life

Identifiers

PMID39758431
PMCPMC11699855

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.