← Назад

Chondrodysplasia-difference of sex development syndrome

ORPHA:1422Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (1)

HHAT
hedgehog acyltransferase
Disease-causing germline mutation(s) (loss of function) in
OMIM: 605743

Фенотипы (22)

Очень частый (80–99%)14
HP:0000252Microcephaly
HP:0000400Macrotia
HP:0000506Telecanthus
HP:0000774Narrow chest
HP:0001249Intellectual disability
HP:0001511Intrauterine growth retardation
HP:0002644Abnormality of pelvic girdle bone morphology
HP:0002983Micromelia
HP:0003043Abnormality of the shoulder
HP:0003510Severe short stature
HP:0004330Increased skull ossification
HP:0005622Broad long bones
HP:0009803Short phalanx of finger
HP:0010049Short metacarpal
Частый (30–79%)8
HP:0000037Male pseudohermaphroditism
HP:0000486Strabismus
HP:0000490Deeply set eye
HP:0000567Chorioretinal coloboma
HP:0000581Blepharophimosis
HP:0000616Miosis
HP:0006872Cerebral hypoplasia
HP:0007676Hypoplasia of the iris

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