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Achondrogenesis type 1A

ORPHA:93299Clinical subtypeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

TRIP11
thyroid hormone receptor interactor 11
Disease-causing germline mutation(s) (loss of function) in
OMIM: 604505

Фенотипы (27)

Очень частый (80–99%)18
HP:0000256Macrocephaly
HP:0000343Long philtrum
HP:0000347Micrognathia
HP:0000463Anteverted nares
HP:0000470Short neck
HP:0000474Thickened nuchal skin fold
HP:0000774Narrow chest
HP:0001789Hydrops fetalis
HP:0002007Frontal bossing
HP:0002983Micromelia
HP:0003196Short nose
HP:0003270Abdominal distention
HP:0003336Abnormal enchondral ossification
HP:0003510Severe short stature
HP:0005716Lethal skeletal dysplasia
HP:0006703Aplasia/Hypoplasia of the lungs
HP:0010306Short thorax
HP:0012368Flat face
Частый (30–79%)7
HP:0001537Umbilical hernia
HP:0001561Polyhydramnios
HP:0001773Short foot
HP:0002757Recurrent fractures
HP:0004279Short palm
HP:0006640Multiple ribs fractures
HP:0100541Femoral hernia
Периодический (5–29%)2
HP:0000476Cystic hygroma
HP:0030680Abnormal cardiovascular system morphology

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