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Potocki-Shaffer syndrome

ORPHA:52022Malformation syndromeUnknownInfancy, Neonatal

Ассоциированные гены (3)

ALX4
ALX homeobox 4
Role in the phenotype of
OMIM: 605420
EXT2
exostosin glycosyltransferase 2
Role in the phenotype of
OMIM: 608210
PHF21A
PHD finger protein 21A
Role in the phenotype of
OMIM: 608325

Фенотипы (23)

Очень частый (80–99%)10
HP:0000248Brachycephaly
HP:0000286Epicanthus
HP:0000347Micrognathia
HP:0000426Prominent nasal bridge
HP:0000430Underdeveloped nasal alae
HP:0000437Depressed nasal tip
HP:0000455Broad nasal tip
HP:0001263Global developmental delay
HP:0004331Decreased skull ossification
HP:0100777Exostoses
Частый (30–79%)7
HP:0000054Micropenis
HP:0000322Short philtrum
HP:0000486Strabismus
HP:0000639Nystagmus
HP:0001250Seizure
HP:0002697Parietal foramina
HP:0002714Downturned corners of mouth
Периодический (5–29%)6
HP:0000821Hypothyroidism
HP:0000822Hypertension
HP:0000823Delayed puberty
HP:0001249Intellectual disability
HP:0001903Anemia
HP:0002667Nephroblastoma

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