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X-linked intellectual disability-hypotonia-facial dysmorphism-aggressive behavior syndrome

ORPHA:85329Malformation syndromeX-linked recessiveChildhood, Infancy

Ассоциированные гены (1)

AP1S2
adaptor related protein complex 1 subunit sigma 2
Disease-causing germline mutation(s) in
OMIM: 300629

Фенотипы (19)

Очень частый (80–99%)16
HP:0000194Open mouth
HP:0000252Microcephaly
HP:0000276Long face
HP:0000325Triangular face
HP:0000331Short chin
HP:0000348High forehead
HP:0000411Protruding ear
HP:0000718Aggressive behavior
HP:0001263Global developmental delay
HP:0001288Gait disturbance
HP:0001290Generalized hypotonia
HP:0001999Abnormal facial shape
HP:0002187Intellectual disability, profound
HP:0003189Long nose
HP:0003202Skeletal muscle atrophy
HP:0011968Feeding difficulties
Частый (30–79%)2
HP:0002353EEG abnormality
HP:0003198Myopathy
Периодический (5–29%)1
HP:0004322Short stature

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