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Terminal osseous dysplasia-pigmentary defects syndrome

ORPHA:88630Malformation syndromeX-linked dominantInfancy, Neonatal

Ассоциированные гены (1)

FLNA
filamin A
Disease-causing germline mutation(s) in
OMIM: 300017

Фенотипы (23)

Очень частый (80–99%)2
HP:0025197Inclusion body fibromatosis
HP:0025473Hyperpigmented papule
Частый (30–79%)5
HP:0000191Accessory oral frenulum
HP:0001156Brachydactyly
HP:0001371Flexion contracture
HP:0001596Alopecia
HP:0030084Clinodactyly
Периодический (5–29%)14
HP:0000286Epicanthus
HP:0000316Hypertelorism
HP:0000437Depressed nasal tip
HP:0000612Iris coloboma
HP:0000685Hypoplasia of teeth
HP:0001159Syndactyly
HP:0001655Patent foramen ovale
HP:0002650Scoliosis
HP:0004322Short stature
HP:0004467Preauricular pit
HP:0009139Osteolysis involving bones of the lower limbs
HP:0010306Short thorax
HP:0012385Camptodactyly
HP:0045039Osteolysis involving bones of the upper limbs
Очень редкий (1–4%)2
HP:0001653Mitral regurgitation
HP:0001723Restrictive cardiomyopathy

Эпидемиология (1)

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