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Laing distal myopathy

ORPHA:59135DiseaseAutosomal dominantChildhood

Ассоциированные гены (1)

MYH7
myosin heavy chain 7
Disease-causing germline mutation(s) in
OMIM: 160760

Фенотипы (20)

Очень частый (80–99%)2
HP:0009027Foot dorsiflexor weakness
HP:0011916Toe extensor amyotrophy
Частый (30–79%)14
HP:0000218High palate
HP:0000467Neck muscle weakness
HP:0001288Gait disturbance
HP:0001430Abnormality of the calf musculature
HP:0002460Distal muscle weakness
HP:0002650Scoliosis
HP:0003323Progressive muscle weakness
HP:0003326Myalgia
HP:0003789Minicore myopathy
HP:0003803Type 1 muscle fiber predominance
HP:0004696Talipes cavus equinovarus
HP:0008180Mildly elevated creatine kinase
HP:0008316Abnormal mitochondria in muscle tissue
HP:0012507Weakness of orbicularis oculi muscle
Периодический (5–29%)3
HP:0001644Dilated cardiomyopathy
HP:0003458EMG: myopathic abnormalities
HP:0008994Proximal muscle weakness in lower limbs
Исключён (0%)1
HP:0003805Rimmed vacuoles

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы