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Thanatophoric dysplasia type 2

ORPHA:93274Clinical subtypeAutosomal dominant, Not applicableInfancy, Neonatal

Ассоциированные гены (1)

FGFR3
fibroblast growth factor receptor 3
Disease-causing germline mutation(s) in
OMIM: 134934

Фенотипы (35)

Очень частый (80–99%)18
HP:0000256Macrocephaly
HP:0000774Narrow chest
HP:0000926Platyspondyly
HP:0000944Abnormal metaphysis morphology
HP:0001156Brachydactyly
HP:0001252Hypotonia
HP:0001582Redundant skin
HP:0002093Respiratory insufficiency
HP:0002652Skeletal dysplasia
HP:0002676Cloverleaf skull
HP:0002983Micromelia
HP:0004322Short stature
HP:0005280Depressed nasal bridge
HP:0006703Aplasia/Hypoplasia of the lungs
HP:0010306Short thorax
HP:0012368Flat face
HP:0100543Cognitive impairment
HP:0001382Joint hypermobility
Частый (30–79%)8
HP:0000365Hearing impairment
HP:0000520Proptosis
HP:0001250Seizure
HP:0001561Polyhydramnios
HP:0002007Frontal bossing
HP:0002119Ventriculomegaly
HP:0002808Kyphosis
HP:0010880Increased nuchal translucency
Периодический (5–29%)9
HP:0000077Abnormality of the kidney
HP:0000238Hydrocephalus
HP:0000956Acanthosis nigricans
HP:0001360Holoprosencephaly
HP:0001376Limitation of joint mobility
HP:0001631Atrial septal defect
HP:0001643Patent ductus arteriosus
HP:0002084Encephalocele
HP:0002269Abnormality of neuronal migration

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы