ArticleNorthern clinics of Istanbul2025
Epidemiological study of congenital myasthenic syndromes based on national electronic health database of Turkiye.
Article in Northern clinics of Istanbul, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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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.
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Who cites it
1 citing paper in PubMed.
- First-in-Human Dose Selection and Safety, Tolerability, Pharmacokinetics, and Immunogenicity of the Muscle-Specific Kinase Agonist Adimanebart (ARGX-119).Journal of clinical pharmacology · 2026Trial
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveCongenital myasthenic syndromes (CMS) represent a group of genetically heterogenous disorders characterized by defective signal transmission at the neuromuscular junction. Although global prevalence of CMS remains uncertain, regional studies have reported varying prevalence rates. This study aimed to define the incidence and prevalence of CMS in Turkiye utilizing data from the national electronic health registry. Additionally, the rate of pyridostigmine prescriptions among patients with CMS was assessed.
methodsThe study was a retrospective national cohort study, and patients with at least three G70.2 ICD-10 code entries between 1 January 2015 and 22 May 2024 were included. While calculating incidence and prevalence rates official census data from the Turkish Statistical Institute were used.
resultsA total of 406 patients were included in the study, with females comprising 48.8% of the cohort. The mean age at diagnosis was 20.59±21.65 years (median: 12.00, min-max: 0-86). Among the cohort, 58.6% were diagnosed before the age of 18, and 12.3% before the age of one. Pyridostigmine was prescribed at least once to 68.2% of the patients. The annual incidence of CMS ranged from 0.28 to 0.59 per million between 2016 and 2023. In 2023, the incidence and prevalence rates of CMS were calculated as 0.63 and 4.49 per million, respectively.
conclusionThis study represents the first comprehensive nationwide epidemiological analysis of CMS in Turkiye utilizing the national electronic health registry. The study enhances the understanding of the epidemiological landscape of CMS in the country by reporting the current incidence, prevalence, and pyridostigmine prescription rates and underscores the significance of this rare but impactful neuromuscular disorder.
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