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MORM syndrome

ORPHA:75858DiseaseAutosomal recessiveAntenatal, Infancy, Neonatal

Ассоциированные гены (1)

INPP5E
inositol polyphosphate-5-phosphatase E
Disease-causing germline mutation(s) in
OMIM: 613037

Фенотипы (18)

Очень частый (80–99%)4
HP:0000054Micropenis
HP:0000505Visual impairment
HP:0000750Delayed speech and language development
HP:0001956Truncal obesity
Частый (30–79%)10
HP:0000518Cataract
HP:0000556Retinal dystrophy
HP:0000718Aggressive behavior
HP:0000752Hyperactivity
HP:0001105Retinal atrophy
HP:0001256Intellectual disability, mild
HP:0002342Intellectual disability, moderate
HP:0002465Poor speech
HP:0007675Progressive night blindness
HP:0010864Intellectual disability, severe
Периодический (5–29%)1
HP:0000077Abnormality of the kidney
Исключён (0%)3
HP:0000613Photophobia
HP:0000639Nystagmus
HP:0001252Hypotonia

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