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RAPADILINO syndrome

ORPHA:3021Malformation syndromeAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

RECQL4
RecQ like helicase 4
Disease-causing germline mutation(s) in
OMIM: 603780

Фенотипы (15)

Очень частый (80–99%)2
HP:0001511Intrauterine growth retardation
HP:0008897Postnatal growth retardation
Частый (30–79%)9
HP:0000175Cleft palate
HP:0000218High palate
HP:0000417Slender nose
HP:0002014Diarrhea
HP:0006498Aplasia/Hypoplasia of the patella
HP:0006501Aplasia/Hypoplasia of the radius
HP:0009601Aplasia/Hypoplasia of the thumb
HP:0011968Feeding difficulties
HP:0040064Abnormality of limbs
Периодический (5–29%)2
HP:0004313Decreased circulating antibody level
HP:0005403Decreased total T cell count
Исключён (0%)2
HP:0001029Poikiloderma
HP:0001249Intellectual disability

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