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X-linked intellectual disability, Hedera type

ORPHA:93952DiseaseX-linked recessiveInfancy

Ассоциированные гены (1)

ATP6AP2
ATPase H+ transporting accessory protein 2
Disease-causing germline mutation(s) in
OMIM: 300556

Фенотипы (31)

Очень частый (80–99%)2
HP:0001249Intellectual disability
HP:0002069Bilateral tonic-clonic seizure
Частый (30–79%)7
HP:0000750Delayed speech and language development
HP:0001270Motor delay
HP:0002317Unsteady gait
HP:0002600Hyporeflexia of lower limbs
HP:0007076Extrapyramidal muscular rigidity
HP:0010819Atonic seizure
HP:0012391Hyporeflexia of upper limbs
Периодический (5–29%)22
HP:0000338Hypomimic face
HP:0001272Cerebellar atrophy
HP:0001288Gait disturbance
HP:0001310Dysmetria
HP:0001350Slurred speech
HP:0001513Obesity
HP:0001621Weak voice
HP:0001712Left ventricular hypertrophy
HP:0001763Pes planus
HP:0001848Calcaneovalgus deformity
HP:0002079Hypoplasia of the corpus callosum
HP:0002186Apraxia
HP:0002307Drooling
HP:0002345Action tremor
HP:0002359Frequent falls
HP:0002540Inability to walk
HP:0002650Scoliosis
HP:0003438Absent Achilles reflex
HP:0003487Babinski sign
HP:0010527Astereognosia
HP:0010529Echolalia
HP:0011812Agraphesthesia

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