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Infantile dystonia-parkinsonism

ORPHA:238455DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (2)

SLC6A3
solute carrier family 6 member 3
Disease-causing germline mutation(s) in
OMIM: 126455
WARS2
tryptophanyl tRNA synthetase 2, mitochondrial
Disease-causing germline mutation(s) in
OMIM: 604733

Фенотипы (20)

Очень частый (80–99%)2
HP:0001300Parkinsonism
HP:0001332Dystonia
Частый (30–79%)18
HP:0000338Hypomimic face
HP:0000737Irritability
HP:0001263Global developmental delay
HP:0001276Hypertonia
HP:0001344Absent speech
HP:0002019Constipation
HP:0002020Gastroesophageal reflux
HP:0002067Bradykinesia
HP:0002072Chorea
HP:0002310Orofacial dyskinesia
HP:0002375Hypokinesia
HP:0002509Limb hypertonia
HP:0004354Abnormality of carboxylic acid metabolism
HP:0007256Abnormal pyramidal sign
HP:0008936Axial hypotonia
HP:0010553Oculogyric crisis
HP:0011968Feeding difficulties
HP:0100021Cerebral palsy

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