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Becker muscular dystrophy

ORPHA:98895DiseaseX-linked recessiveAdolescent, Adult, Childhood, Elderly

Ассоциированные гены (1)

DMD
dystrophin
Disease-causing germline mutation(s) in
OMIM: 300377

Фенотипы (16)

Очень частый (80–99%)7
HP:0001288Gait disturbance
HP:0002913Myoglobinuria
HP:0003236Elevated circulating creatine kinase concentration
HP:0003326Myalgia
HP:0003546Exercise intolerance
HP:0003551Difficulty climbing stairs
HP:0012086Abnormal urinary color
Частый (30–79%)6
HP:0001324Muscle weakness
HP:0002527Falls
HP:0002814Abnormality of the lower limb
HP:0002910Elevated circulating hepatic transaminase concentration
HP:0003394Muscle spasm
HP:0012378Fatigue
Периодический (5–29%)3
HP:0001763Pes planus
HP:0003202Skeletal muscle atrophy
HP:0030051Tip-toe gait

Эпидемиология (13)

Point prevalence
1-9 / 100 000
Europe
Prevalence at birth
1-9 / 100 000
United Kingdom
Point prevalence
1-9 / 100 000
United Kingdom
Prevalence at birth
1-9 / 100 000
United States
Point prevalence
1-9 / 100 000
United States
Prevalence at birth
1-9 / 100 000
Europe
Point prevalence
1-9 / 100 000
Worldwide
Point prevalence
1-9 / 100 000
Italy
Point prevalence
1-9 / 100 000
Egypt
Point prevalence
1-9 / 1 000 000
Japan
Point prevalence
<1 / 1 000 000
South Africa
Point prevalence
1-9 / 100 000
Ireland
Point prevalence
1-9 / 100 000
Puerto rico

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы