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Cerebellar ataxia-hypogonadism syndrome

ORPHA:1173DiseaseAutosomal recessiveAdolescent, Adult, Childhood

Ассоциированные гены (2)

PNPLA6
patatin like domain 6, lysophospholipase
Disease-causing germline mutation(s) in
OMIM: 603197
RNF216
ring finger protein 216
Disease-causing germline mutation(s) (loss of function) in
OMIM: 609948

Фенотипы (20)

Очень частый (80–99%)11
HP:0000044Hypogonadotropic hypogonadism
HP:0000135Hypogonadism
HP:0000144Decreased fertility
HP:0000512Abnormal electroretinogram
HP:0000639Nystagmus
HP:0000648Optic atrophy
HP:0000771Gynecomastia
HP:0000864Abnormality of the hypothalamus-pituitary axis
HP:0001251Ataxia
HP:0002167Abnormality of speech or vocalization
HP:0007703Abnormality of retinal pigmentation
Частый (30–79%)2
HP:0001252Hypotonia
HP:0004374Hemiplegia/hemiparesis
Периодический (5–29%)7
HP:0000248Brachycephaly
HP:0000708Atypical behavior
HP:0000726Dementia
HP:0000751Personality changes
HP:0002558Supernumerary nipple
HP:0004209Clinodactyly of the 5th finger
HP:0004322Short stature

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