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Autosomal dominant spastic paraplegia type 73

ORPHA:444099DiseaseAutosomal dominantAdult

Ассоциированные гены (1)

CPT1C
carnitine palmitoyltransferase 1C
Disease-causing germline mutation(s) in
OMIM: 608846

Фенотипы (19)

Очень частый (80–99%)6
HP:0001347Hyperreflexia
HP:0002061Lower limb spasticity
HP:0002064Spastic gait
HP:0002314Degeneration of the lateral corticospinal tracts
HP:0003487Babinski sign
HP:0007020Progressive spastic paraplegia
Частый (30–79%)9
HP:0000012Urinary urgency
HP:0000020Urinary incontinence
HP:0001288Gait disturbance
HP:0002166Impaired vibration sensation in the lower limbs
HP:0003457EMG abnormality
HP:0007199Progressive spastic paraparesis
HP:0008944Distal lower limb amyotrophy
HP:0009053Distal lower limb muscle weakness
HP:0012898Abnormal lower-limb motor evoked potentials
Периодический (5–29%)1
HP:0001761Pes cavus
Исключён (0%)3
HP:0000726Dementia
HP:0001250Seizure
HP:0002921Abnormality of the cerebrospinal fluid

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
<1 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы