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Roussy-Lévy syndrome

ORPHA:3115DiseaseAutosomal dominantChildhood, Infancy

Ассоциированные гены (2)

PMP22
peripheral myelin protein 22
Disease-causing germline mutation(s) in
OMIM: 601097
MPZ
myelin protein zero
Disease-causing germline mutation(s) in
OMIM: 159440

Фенотипы (27)

Частый (30–79%)21
HP:0001288Gait disturbance
HP:0000639Nystagmus
HP:0001284Areflexia
HP:0001761Pes cavus
HP:0001762Talipes equinovarus
HP:0002066Gait ataxia
HP:0002070Limb ataxia
HP:0002174Postural tremor
HP:0002312Clumsiness
HP:0002317Unsteady gait
HP:0002495Impaired vibratory sensation
HP:0002650Scoliosis
HP:0002751Kyphoscoliosis
HP:0003431Decreased motor nerve conduction velocity
HP:0003474Somatic sensory dysfunction
HP:0003693Distal amyotrophy
HP:0007131Acute demyelinating polyneuropathy
HP:0007141Sensorimotor neuropathy
HP:0007328Impaired pain sensation
HP:0007340Lower limb muscle weakness
HP:0010829Impaired temperature sensition
Периодический (5–29%)6
HP:0002359Frequent falls
HP:0002839Urinary bladder sphincter dysfunction
HP:0002857Genu valgum
HP:0003202Skeletal muscle atrophy
HP:0003487Babinski sign
HP:0008954Intrinsic hand muscle atrophy

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