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Noonan syndrome-like disorder with loose anagen hair

ORPHA:2701Malformation syndromeAutosomal dominantAntenatal, Infancy, Neonatal

Ассоциированные гены (2)

PPP1CB
protein phosphatase 1 catalytic subunit beta
Disease-causing germline mutation(s) in
OMIM: 600590
SHOC2
SHOC2 leucine rich repeat scaffold protein
Disease-causing germline mutation(s) in
OMIM: 602775

Фенотипы (29)

Очень частый (80–99%)7
HP:0000465Webbed neck
HP:0002162Low posterior hairline
HP:0002209Sparse scalp hair
HP:0002750Delayed skeletal maturation
HP:0003196Short nose
HP:0004322Short stature
HP:0000358Posteriorly rotated ears
Частый (30–79%)9
HP:0000238Hydrocephalus
HP:0000286Epicanthus
HP:0000400Macrotia
HP:0000463Anteverted nares
HP:0000767Pectus excavatum
HP:0001639Hypertrophic cardiomyopathy
HP:0001642Pulmonic stenosis
HP:0002002Deep philtrum
HP:0100840Aplasia/Hypoplasia of the eyebrow
Периодический (5–29%)13
HP:0000028Cryptorchidism
HP:0000174Abnormal palate morphology
HP:0000179Thick lower lip vermilion
HP:0000233Thin vermilion border
HP:0000316Hypertelorism
HP:0000365Hearing impairment
HP:0000670Carious teeth
HP:0001156Brachydactyly
HP:0001231Abnormal fingernail morphology
HP:0001249Intellectual disability
HP:0001800Hypoplastic toenails
HP:0005108Abnormal intervertebral disk morphology
HP:0009811Abnormality of the elbow

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