Article in Neurology. Genetics, 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.
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
21 authors.
Meredith K JamesJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0000-0001-8078-7744
Dionne MoatJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0009-0000-9829-7647
Jassi Michell-SodhiJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0009-0000-3877-3824
Karen S W WongJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0009-0000-1928-0469
Emma GroverJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0009-0008-7689-086X
Emma-Jayne RobinsonJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0000-0002-6221-4691
Anna G MayhewJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0000-0001-7085-9011
Maha ElseedJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.
Michela GuglieriJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0000-0002-8455-0637
Volker StraubJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0000-0001-9046-3540
Chiara Marini-BettoloJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0000-0003-3795-6017
Robert Muni-LofraJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0000-0002-8901-9140
Jordi Diaz-ManeraJohn Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, The Newcastle upon Tyne Hospitals NHS Trust and Newcastle University, United Kingdom.ORCID https://orcid.org/0000-0003-2941-7988
Background and Objectives: Limb-girdle muscular dystrophy (LGMD) type R1/2A, calpain-3-related, is a rare, autosomal recessive disorder caused by pathogenic variants in the Methods: Ninety-two individuals were evaluated by physiotherapy teams at Nationwide Children's Hospital and the John Walton Muscular Dystrophy Research Centre. Psychometric analysis of NSAD and PUL was completed using Rasch measurement methods. Descriptive statistics and correlation coefficients explored the relationship among all outcomes. Results: Psychometric analyses demonstrated that NSAD and PUL were valid and appropriate functional outcome measures for ambulant and nonambulant individuals with LGMDR1/2A. The NSAD and 100MTT velocity were highly correlated (Spearman rho Rs(88) = 0.89) The 100MTT was particularly useful for the strongest and asymptomatic cohort. Discussion: In this largest reported cohort of individuals with LGMDR1/2A, NSAD, PUL, and 100MTT were suitable to quantify functional impact of the disease. International harmonization of outcome measures creates meaningful clinical data to inform clinical management and trial design.
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.
Motor Function in Limb-Girdle Muscular Dystrophy R1/2A: Validation of Clinical Outcome Assessments for Clinical Care and Trial Readiness. · full record | OpenQuestion