ArticleJIMD reports2025
Treatment Beliefs Reflect Unmet Clinical Needs in Lysosomal Storage Diseases: An Opportunity for a Patient-Centered Approach.
Article in JIMD reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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.
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.
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Authors and funding
5 authors.
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No grant is acknowledged in the PubMed record.
Abstract
Despite life-long pharmacotherapy for many people affected by lysosomal storage diseases, no data are available on their beliefs about their treatments. Therapeutic options range from disease-specific, with varying levels of effectiveness, to purely supportive. This spectrum is illustrated by the three diseases Gaucher disease type 1 (effective disease-specific therapies), Fabry disease (disease-specific therapies with variable effectiveness), and mucopolysaccharidosis type III A/B (supportive care only). Employing the Necessity-Concerns Framework of the Beliefs in Medicine Questionnaire, we investigated intra- and intergroup variability in adults with Gaucher disease type 1, Fabry disease, and parents of children with mucopolysaccharidosis type III A/B. Participants rated
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Registered trials
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 Socratic graph.