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X-linked intellectual disability-hypotonia-movement disorder syndrome

ORPHA:457260DiseaseX-linked dominantAdolescent, Childhood

Ассоциированные гены (1)

DDX3X
DEAD-box helicase 3 X-linked
Disease-causing germline mutation(s) (loss of function) in
OMIM: 300160

Фенотипы (23)

Облигатный (100%)1
HP:0001249Intellectual disability
Частый (30–79%)9
HP:0000252Microcephaly
HP:0000504Abnormality of vision
HP:0000718Aggressive behavior
HP:0000729Autistic behavior
HP:0000752Hyperactivity
HP:0001257Spasticity
HP:0001290Generalized hypotonia
HP:0002136Broad-based gait
HP:0100660Dyskinesia
Периодический (5–29%)13
HP:0000202Orofacial cleft
HP:0000365Hearing impairment
HP:0000505Visual impairment
HP:0000826Precocious puberty
HP:0001000Abnormality of skin pigmentation
HP:0001250Seizure
HP:0001263Global developmental delay
HP:0001382Joint hypermobility
HP:0001999Abnormal facial shape
HP:0002079Hypoplasia of the corpus callosum
HP:0002119Ventriculomegaly
HP:0002539Cortical dysplasia
HP:0002650Scoliosis

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