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X-linked adrenal hypoplasia congenita

ORPHA:95702DiseaseX-linked recessiveChildhood, Infancy

Ассоциированные гены (1)

NR0B1
nuclear receptor subfamily 0 group B member 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 300473

Фенотипы (24)

Облигатный (100%)1
HP:0003154Increased circulating ACTH level
Очень частый (80–99%)2
HP:0000953Hyperpigmentation of the skin
HP:0008163Decreased circulating cortisol level
Частый (30–79%)18
HP:0040171Decreased serum testosterone concentration
HP:0046504Decreased libido
HP:0100639Erectile dysfunction
HP:0000044Hypogonadotropic hypogonadism
HP:0000798Oligozoospermia
HP:0000823Delayed puberty
HP:0001531Failure to thrive in infancy
HP:0002013Vomiting
HP:0002014Diarrhea
HP:0002018Nausea
HP:0002153Hyperkalemia
HP:0002321Vertigo
HP:0002902Hyponatremia
HP:0008186Adrenocortical cytomegaly
HP:0008207Primary adrenal insufficiency
HP:0008734Decreased testicular size
HP:0012378Fatigue
HP:0030344Decreased circulating luteinizing hormone level
Периодический (5–29%)3
HP:0001250Seizure
HP:0001824Weight loss
HP:0002225Sparse pubic hair

Эпидемиология (2)

Point prevalence
1-9 / 100 000
Worldwide
Prevalence at birth
1-9 / 100 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы