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Paroxysmal exertion-induced dyskinesia

ORPHA:98811DiseaseAutosomal dominant, Not applicableAdolescent, Adult, Childhood, Infancy

Ассоциированные гены (2)

SLC2A1
solute carrier family 2 member 1
Disease-causing germline mutation(s) in
OMIM: 138140
PRRT2
proline rich transmembrane protein 2
Disease-causing germline mutation(s) in
OMIM: 614386

Фенотипы (17)

Очень частый (80–99%)3
HP:0001266Choreoathetosis
HP:0001332Dystonia
HP:0007166Paroxysmal dyskinesia
Частый (30–79%)7
HP:0001249Intellectual disability
HP:0001250Seizure
HP:0001304Torsion dystonia
HP:0002121Generalized non-motor (absence) seizure
HP:0003401Paresthesia
HP:0004305Involuntary movements
HP:0006801Hyperactive deep tendon reflexes
Периодический (5–29%)5
HP:0000718Aggressive behavior
HP:0000737Irritability
HP:0001251Ataxia
HP:0001328Specific learning disability
HP:0002072Chorea
Очень редкий (1–4%)2
HP:0001256Intellectual disability, mild
HP:0002061Lower limb spasticity

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