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Alopecia-intellectual disability syndrome

ORPHA:2850DiseaseAutosomal recessiveNeonatal

Ассоциированные гены (3)

ITGB6
integrin subunit beta 6
Disease-causing germline mutation(s) in
OMIM: 147558
LSS
lanosterol synthase
Disease-causing germline mutation(s) in
OMIM: 600909
AHSG
alpha 2-HS glycoprotein
Disease-causing germline mutation(s) in
OMIM: 138680

Фенотипы (26)

Очень частый (80–99%)9
HP:0000252Microcephaly
HP:0000365Hearing impairment
HP:0001249Intellectual disability
HP:0001252Hypotonia
HP:0001596Alopecia
HP:0002209Sparse scalp hair
HP:0002231Sparse body hair
HP:0002750Delayed skeletal maturation
HP:0100840Aplasia/Hypoplasia of the eyebrow
Частый (30–79%)12
HP:0000613Photophobia
HP:0000815Hypergonadotropic hypogonadism
HP:0001156Brachydactyly
HP:0001171Split hand
HP:0001250Seizure
HP:0001510Growth delay
HP:0002353EEG abnormality
HP:0004322Short stature
HP:0008064Ichthyosis
HP:0008070Sparse hair
HP:0011842Abnormality of skeletal morphology
HP:0200012Short corpus callosum
Периодический (5–29%)5
HP:0000400Macrotia
HP:0000962Hyperkeratosis
HP:0001371Flexion contracture
HP:0002650Scoliosis
HP:0005105Abnormal nasal morphology

Эпидемиология (2)

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

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы