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Hereditary neuropathy with liability to pressure palsies

ORPHA:640Malformation syndromeAutosomal dominantAdolescent, Adult, Childhood, Elderly, Infancy

Ассоциированные гены (2)

PMP22
peripheral myelin protein 22
Disease-causing germline mutation(s) in
OMIM: 601097
PMP22
peripheral myelin protein 22
Role in the phenotype of
OMIM: 601097

Фенотипы (18)

Очень частый (80–99%)2
HP:0003431Decreased motor nerve conduction velocity
HP:0009830Peripheral neuropathy
Частый (30–79%)5
HP:0001324Muscle weakness
HP:0002650Scoliosis
HP:0003401Paresthesia
HP:0007141Sensorimotor neuropathy
HP:0200101Decreased/absent ankle reflexes
Периодический (5–29%)11
HP:0000407Sensorineural hearing impairment
HP:0001265Hyporeflexia
HP:0001605Vocal cord paralysis
HP:0001608Abnormality of the voice
HP:0001761Pes cavus
HP:0002093Respiratory insufficiency
HP:0003394Muscle spasm
HP:0003693Distal amyotrophy
HP:0003704Scapuloperoneal weakness
HP:0003738Exercise-induced myalgia
HP:0006824Cranial nerve paralysis

Эпидемиология (2)

Point prevalence
1-9 / 100 000
Europe
Point prevalence
1-5 / 10 000
Finland

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы