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Congenital adrenal hyperplasia due to 11-beta-hydroxylase deficiency

ORPHA:90795DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

CYP11B1
cytochrome P450 family 11 subfamily B member 1
Disease-causing germline mutation(s) in
OMIM: 610613

Фенотипы (32)

Очень частый (80–99%)11
HP:0000062Ambiguous genitalia
HP:0000822Hypertension
HP:0001507Growth abnormality
HP:0002900Hypokalemia
HP:0003351Decreased circulating renin concentration
HP:0005616Accelerated skeletal maturation
HP:0008163Decreased circulating cortisol level
HP:0025380Increased circulating androstenedione concentration
HP:0030348Increased circulating androgen concentration
HP:0031213Elevated circulating 17-hydroxyprogesterone
HP:0032330Increased urinary 11-deoxycorticosterone level
Частый (30–79%)13
HP:0000040Long penis
HP:0000061Ambiguous genitalia, female
HP:0000826Precocious puberty
HP:0000858Irregular menstruation
HP:0000953Hyperpigmentation of the skin
HP:0001007Hirsutism
HP:0001061Acne
HP:0003154Increased circulating ACTH level
HP:0004322Short stature
HP:0008236Isosexual precocious puberty
HP:0008665Clitoral hypertrophy
HP:0012411Premature pubarche
HP:0012412Premature adrenarche
Периодический (5–29%)6
HP:0000127Renal salt wasting
HP:0000147Polycystic ovaries
HP:0000771Gynecomastia
HP:0001596Alopecia
HP:0025451Testicular adrenal rest tumor
HP:0030088Increased serum testosterone level
Очень редкий (1–4%)2
HP:0002170Intracranial hemorrhage
HP:0010314Premature thelarche

Эпидемиология (2)

Prevalence at birth
1-9 / 1 000 000
Europe
Point prevalence
1-9 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы