ArticleJIMD reports2020
Morquio-B disease: Clinical and genetic characteristics of a distinct
Article in JIMD reports, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
15 citing papers in PubMed, 21 citations in OpenAlex.
- Review of clinical trials and guidelines for children and youth with mucopolysaccharidosis: outcome selection and measurement.Orphanet journal of rare diseases · 2024Review
- AMolecular therapy. Methods & clinical development · 2024Article
- Congenital Heart Malformations Masked by Infantile Gangliosidosis-Case Report and Growing Evidence for Metabolic Disease-Associated Aortopathies.Diagnostics (Basel, Switzerland) · 2024Article
- Morquio B disease: a case report.Frontiers in pediatrics · 2024Article
- Validation of a highly sensitive HaloTag-based assay to evaluate the potency of a novel class of allosteric β-Galactosidase correctors.PloS one · 2023Article
- Common protein-coding variants influence the racing phenotype in galloping racehorse breeds.Communications biology · 2022Article
- β-Galactosidase deficiency in the GLB1 spectrum of lysosomal storage disease can present with severe muscle weakness and atrophy.JIMD reports · 2022Article
- Lysosomal positioning diseases: beyond substrate storage.Open biology · 2022Review
- Processed pseudogene insertion in GLB1 causes Morquio B disease by altering intronic splicing regulatory landscape.NPJ genomic medicine · 2022Article
- Morquio-like dysostosis multiplex presenting with neuronopathic features is a distinctJIMD reports · 2021Article
- Are GMI gangliosidosis and Morquio type B two different disorders or part of one phenotypic spectrum?JIMD reports · 2021Article
- Epidemiology of Mucopolysaccharidoses Update.Diagnostics (Basel, Switzerland) · 2021Review
- GM1 Gangliosidosis-A Mini-Review.Frontiers in genetics · 2021Review
- Morquio B Disease. Disease Characteristics and Treatment Options of a DistinctInternational journal of molecular sciences · 2020Review
- Morquio-B disease: Clinical and genetic characteristics of a distinctJIMD reports · 2020Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundMorquio-B disease (MBD) is a distinct
methodsWe analyzed 63 (n = 62 published) cases with MBD to describe their clinical, biochemical and genetic features.
resultsForty-one of 51 cases with informative clinical data had
conclusionData provide a basis for future systematic collection of clinical, biochemical, morphologic, and genetic data of this ultra-rare condition.
Indexed as
Identifiers
What OpenQuestion holds
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 OpenQuestion graph.