← Назад

Adult-onset chronic progressive external ophthalmoplegia with mitochondrial myopathy

ORPHA:329336DiseaseAutosomal dominant, Mitochondrial inheritanceAdult

Ассоциированные гены (2)

RRM2B
ribonucleotide reductase regulatory TP53 inducible subunit M2B
Disease-causing germline mutation(s) in
OMIM: 604712
RNASEH1
ribonuclease H1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 604123

Фенотипы (32)

Облигатный (100%)1
HP:0000590Progressive external ophthalmoplegia
Очень частый (80–99%)2
HP:0003546Exercise intolerance
HP:0003690Limb muscle weakness
Частый (30–79%)14
HP:0000218High palate
HP:0000365Hearing impairment
HP:0001260Dysarthria
HP:0001488Bilateral ptosis
HP:0002015Dysphagia
HP:0002522Areflexia of lower limbs
HP:0002747Respiratory insufficiency due to muscle weakness
HP:0003202Skeletal muscle atrophy
HP:0003326Myalgia
HP:0003551Difficulty climbing stairs
HP:0003738Exercise-induced myalgia
HP:0011968Feeding difficulties
HP:0030196Fatigable weakness of respiratory muscles
HP:0030319Weakness of facial musculature
Периодический (5–29%)15
HP:0000565Esotropia
HP:0000580Pigmentary retinopathy
HP:0001638Cardiomyopathy
HP:0002076Migraine
HP:0002141Gait imbalance
HP:0002361Psychomotor deterioration
HP:0002406Limb dysmetria
HP:0002505Loss of ambulation
HP:0002549Deficit in phonologic short-term memory
HP:0002650Scoliosis
HP:0003133Abnormality of the spinocerebellar tracts
HP:0003722Neck flexor weakness
HP:0005150Abnormal atrioventricular conduction
HP:0007141Sensorimotor neuropathy
HP:0007256Abnormal pyramidal sign

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