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Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency

ORPHA:90794DiseaseAutosomal recessiveAdolescent, Adult, Antenatal, Childhood, Infancy, Neonatal

Фенотипы (56)

Очень частый (80–99%)5
HP:0008163Decreased circulating cortisol level
HP:0030348Increased circulating androgen concentration
HP:0031074Abnormal response to ACTH stimulation test
HP:0031213Elevated circulating 17-hydroxyprogesterone
HP:0031216Increased circulating progesterone
Частый (30–79%)36
HP:0000127Renal salt wasting
HP:0000140Abnormality of the menstrual cycle
HP:0000144Decreased fertility
HP:0000811Abnormal external genitalia
HP:0000841Hyperactive renin-angiotensin system
HP:0000876Oligomenorrhea
HP:0001007Hirsutism
HP:0001061Acne
HP:0001507Growth abnormality
HP:0001508Failure to thrive
HP:0001824Weight loss
HP:0001944Dehydration
HP:0001998Neonatal hypoglycemia
HP:0002013Vomiting
HP:0002153Hyperkalemia
HP:0002615Hypotension
HP:0002902Hyponatremia
HP:0003113Hypochloremia
HP:0003154Increased circulating ACTH level
HP:0003639Elevated urinary epinephrine
HP:0004012Premature fusion of the radial epiphyseal plates
HP:0004319Decreased circulating aldosterone level
HP:0004322Short stature
HP:0005616Accelerated skeletal maturation
HP:0005976Hyperkalemic metabolic acidosis
HP:0008207Primary adrenal insufficiency
HP:0008665Clitoral hypertrophy
HP:0011106Hypovolemia
HP:0011968Feeding difficulties
HP:0012411Premature pubarche
HP:0012417Hypocapnia
HP:0012605Hypernatriuria
HP:0025380Increased circulating androstenedione concentration
HP:0030088Increased serum testosterone level
HP:0031066Abnormal ovarian physiology
HP:0500022Abnormal serum dehydroepiandrosterone level
Периодический (5–29%)15
HP:0000040Long penis
HP:0000044Hypogonadotropic hypogonadism
HP:0000061Ambiguous genitalia, female
HP:0000062Ambiguous genitalia
HP:0000098Tall stature
HP:0000151Aplasia of the uterus
HP:0002292Frontal balding
HP:0005268Spontaneous abortion
HP:0008734Decreased testicular size
HP:0010465Precocious puberty in females
HP:0012412Premature adrenarche
HP:0025451Testicular adrenal rest tumor
HP:0025486Fused labia majora
HP:0031273Shock
HP:0100779Urogenital sinus anomaly

Эпидемиология (11)

Point prevalence
1-9 / 100 000
Europe
Prevalence at birth
1-9 / 100 000
Worldwide
Prevalence at birth
1-9 / 100 000
France
Prevalence at birth
1-9 / 100 000
Italy
Prevalence at birth
1-9 / 100 000
Sweden
Prevalence at birth
1-9 / 100 000
United States
Prevalence at birth
1-9 / 100 000
New Zealand
Prevalence at birth
1-9 / 100 000
Croatia
Prevalence at birth
1-9 / 100 000
Estonia
Prevalence at birth
1-9 / 100 000
Singapore
Prevalence at birth
1-9 / 100 000
Switzerland

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы