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Congenital adrenal hyperplasia due to 3-beta-hydroxysteroid dehydrogenase deficiency

ORPHA:90791DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

HSD3B2
hydroxy-delta-5-steroid dehydrogenase, 3 beta- and steroid delta-isomerase 2
Disease-causing germline mutation(s) in
OMIM: 613890

Фенотипы (36)

Очень частый (80–99%)1
HP:0008258Congenital adrenal hyperplasia
Частый (30–79%)23
HP:0000033Ambiguous genitalia, male
HP:0000047Hypospadias
HP:0000061Ambiguous genitalia, female
HP:0000127Renal salt wasting
HP:0000771Gynecomastia
HP:0000848Increased circulating renin level
HP:0000953Hyperpigmentation of the skin
HP:0001944Dehydration
HP:0001998Neonatal hypoglycemia
HP:0002013Vomiting
HP:0002153Hyperkalemia
HP:0002615Hypotension
HP:0002902Hyponatremia
HP:0004319Decreased circulating aldosterone level
HP:0008163Decreased circulating cortisol level
HP:0008665Clitoral hypertrophy
HP:0011749Adrenocorticotropic hormone excess
HP:0012041Decreased fertility in males
HP:0025380Increased circulating androstenedione concentration
HP:0030088Increased serum testosterone level
HP:0031213Elevated circulating 17-hydroxyprogesterone
HP:0040171Decreased serum testosterone concentration
HP:0500022Abnormal serum dehydroepiandrosterone level
Периодический (5–29%)12
HP:0000027Azoospermia
HP:0000028Cryptorchidism
HP:0000037Male pseudohermaphroditism
HP:0000808Penoscrotal hypospadias
HP:0001007Hirsutism
HP:0001263Global developmental delay
HP:0001508Failure to thrive
HP:0008730Female external genitalia in individual with 46,XY karyotype
HP:0008734Decreased testicular size
HP:0012412Premature adrenarche
HP:0012768Neonatal asphyxia
HP:0012881Abnormality of the labia majora

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