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X-linked dominant chondrodysplasia, Chassaing-Lacombe type

ORPHA:163966DiseaseX-linked dominantInfancy, Neonatal

Ассоциированные гены (1)

HDAC6
histone deacetylase 6
Disease-causing germline mutation(s) in
OMIM: 300272

Фенотипы (24)

Очень частый (80–99%)14
HP:0000238Hydrocephalus
HP:0000369Low-set ears
HP:0000457Depressed nasal ridge
HP:0000568Microphthalmia
HP:0000926Platyspondyly
HP:0001773Short foot
HP:0002007Frontal bossing
HP:0002866Hypoplastic iliac wing
HP:0003196Short nose
HP:0004279Short palm
HP:0004322Short stature
HP:0005871Metaphyseal chondrodysplasia
HP:0006028Metaphyseal cupping of metacarpals
HP:0008905Rhizomelia
Частый (30–79%)5
HP:0000883Thin ribs
HP:0001256Intellectual disability, mild
HP:0001511Intrauterine growth retardation
HP:0001522Death in infancy
HP:0008364Abnormality of the calcaneus
Периодический (5–29%)5
HP:0000154Wide mouth
HP:0000322Short philtrum
HP:0000347Micrognathia
HP:0000962Hyperkeratosis
HP:0001321Cerebellar hypoplasia

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