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Thomsen and Becker disease

ORPHA:614DiseaseAutosomal dominant, Autosomal recessiveAdolescent, Adult, Childhood, Infancy

Ассоциированные гены (1)

CLCN1
chloride voltage-gated channel 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 118425

Фенотипы (15)

Очень частый (80–99%)5
HP:0002486Myotonia
HP:0003457EMG abnormality
HP:0003552Muscle stiffness
HP:0003740Myotonia with warm-up phenomenon
HP:0100284EMG: myotonic discharges
Частый (30–79%)2
HP:0003326Myalgia
HP:0003712Skeletal muscle hypertrophy
Периодический (5–29%)5
HP:0002312Clumsiness
HP:0003394Muscle spasm
HP:0003458EMG: myopathic abnormalities
HP:0008872Feeding difficulties in infancy
HP:0009063Progressive distal muscle weakness
Очень редкий (1–4%)3
HP:0002015Dysphagia
HP:0011675Arrhythmia
HP:0031546Cardiac conduction abnormality

Эпидемиология (6)

Point prevalence
1-9 / 100 000
Worldwide
Point prevalence
1-9 / 100 000
Europe
Point prevalence
1-9 / 100 000
Finland
Point prevalence
1-9 / 1 000 000
Italy
Point prevalence
1-9 / 1 000 000
United Kingdom
Point prevalence
1-9 / 1 000 000
Netherlands

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы