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Longitudinal analysis of a patient-reported outcome (ACTIVLIM) in Duchenne muscular dystrophy : a registry-based study
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Overview of the Belgian neuromuscular diseases registry : more than a decade of data
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Long-term safety and efficacy of nipocalimab in generalized myasthenia gravis : vivacity-MG3 open-label extension phase results
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Phenotypical and genotypical characterization of a cohort of 196 COL6-RD patients in Europe and Chile
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Efficacy of SMA NBS : 4-year comparative study with control group
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272nd ENMC international workshop : 10 years of progress : revision of the ENMC 2013 diagnostic criteria for inclusion body myositis and clinical trial readiness. 16-18 June 2023, Hoofddorp, The Netherlands
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Large-scale proteomics profiling of peripheral blood of DM1 patients identifies biomarkers for disease severity and physical activity
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- Journal Article
- A1
- open access
Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6-48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
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- Journal Article
- A1
- open access
Risk of malignant hyperthermia in patients carrying a variant in the skeletal muscle ryanodine receptor 1 gene
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239th ENMC International Workshop : Classification of dermatomyositis, Amsterdam, the Netherlands, 14-16 December 2018