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Dopamine beta-hydroxylase deficiency

ORPHA:230DiseaseAutosomal recessiveAdolescent, Adult, Childhood, Infancy, Neonatal

Ассоциированные гены (1)

DBH
dopamine beta-hydroxylase
Disease-causing germline mutation(s) in
OMIM: 609312

Фенотипы (28)

Очень частый (80–99%)4
HP:0001278Orthostatic hypotension
HP:0001488Bilateral ptosis
HP:0011979Elevated urinary dopamine
HP:0012384Rhinitis
Частый (30–79%)10
HP:0001279Syncope
HP:0001315Reduced tendon reflexes
HP:0001903Anemia
HP:0001943Hypoglycemia
HP:0002360Sleep abnormality
HP:0003138Increased blood urea nitrogen
HP:0003259Elevated circulating creatinine concentration
HP:0009020Exercise-induced muscle fatigue
HP:0012378Fatigue
HP:0012877Retrograde ejaculation
Периодический (5–29%)12
HP:0000017Nocturia
HP:0000622Blurred vision
HP:0001252Hypotonia
HP:0001944Dehydration
HP:0002013Vomiting
HP:0002014Diarrhea
HP:0002045Hypothermia
HP:0002094Dyspnea
HP:0002321Vertigo
HP:0003115Abnormal EKG
HP:0012670Orthostatic syncope
HP:0100749Chest pain
Очень редкий (1–4%)2
HP:0000842Hyperinsulinemia
HP:0000855Insulin resistance

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