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Donohue syndrome

ORPHA:508Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

INSR
insulin receptor
Disease-causing germline mutation(s) in
OMIM: 147670

Фенотипы (47)

Очень частый (80–99%)8
HP:0000855Insulin resistance
HP:0000962Hyperkeratosis
HP:0000998Hypertrichosis
HP:0003162Fasting hypoglycemia
HP:0003758Reduced subcutaneous adipose tissue
HP:0008846Severe intrauterine growth retardation
HP:0008897Postnatal growth retardation
HP:0011998Postprandial hyperglycemia
Частый (30–79%)18
HP:0000040Long penis
HP:0000065Labial hypertrophy
HP:0000105Enlarged kidney
HP:0000842Hyperinsulinemia
HP:0000956Acanthosis nigricans
HP:0001249Intellectual disability
HP:0001508Failure to thrive
HP:0001639Hypertrophic cardiomyopathy
HP:0002219Facial hypertrichosis
HP:0002240Hepatomegaly
HP:0003202Skeletal muscle atrophy
HP:0003247Overgrowth of external genitalia
HP:0003270Abdominal distention
HP:0004325Decreased body weight
HP:0004405Prominent nipples
HP:0004914Recurrent infantile hypoglycemia
HP:0008665Clitoral hypertrophy
HP:0011344Severe global developmental delay
Периодический (5–29%)21
HP:0000121Nephrocalcinosis
HP:0000252Microcephaly
HP:0000307Pointed chin
HP:0000316Hypertelorism
HP:0000369Low-set ears
HP:0000411Protruding ear
HP:0000445Wide nose
HP:0000848Increased circulating renin level
HP:0000859Hyperaldosteronism
HP:0000974Hyperextensible skin
HP:0001072Thickened skin
HP:0001176Large hands
HP:0001833Long foot
HP:0002035Rectal prolapse
HP:0002150Hypercalciuria
HP:0002900Hypokalemia
HP:0008936Axial hypotonia
HP:0011787Central hypothyroidism
HP:0012471Thick vermilion border
HP:0025024Megarectum
HP:0100879Enlarged ovaries

Эпидемиология (3)

Point prevalence
<1 / 1 000 000
Worldwide
Point prevalence
<1 / 1 000 000
Europe
Prevalence at birth
<1 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы