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Isolated Pierre Robin sequence

ORPHA:718Malformation syndromeAutosomal dominant, Multigenic/multifactorial, Not applicable, UnknownAntenatal, Neonatal

Ассоциированные гены (1)

SOX9
SRY-box transcription factor 9
Disease-causing germline mutation(s) in
OMIM: 608160

Фенотипы (21)

Очень частый (80–99%)3
HP:0000162Glossoptosis
HP:0000175Cleft palate
HP:0000347Micrognathia
Частый (30–79%)6
HP:0001508Failure to thrive
HP:0001561Polyhydramnios
HP:0002643Neonatal respiratory distress
HP:0002781Upper airway obstruction
HP:0008872Feeding difficulties in infancy
HP:0012418Hypoxemia
Периодический (5–29%)12
HP:0000453Choanal atresia
HP:0000961Cyanosis
HP:0001601Laryngomalacia
HP:0001607Subglottic stenosis
HP:0001648Cor pulmonale
HP:0002015Dysphagia
HP:0002777Tracheal stenosis
HP:0002779Tracheomalacia
HP:0002780Bronchomalacia
HP:0004890Elevated pulmonary artery pressure
HP:0010307Stridor
HP:0010535Sleep apnea

Эпидемиология (15)

Point prevalence
1-9 / 100 000
United Kingdom
Prevalence at birth
1-9 / 100 000
France
Point prevalence
1-9 / 100 000
France
Prevalence at birth
1-5 / 10 000
Germany
Prevalence at birth
1-9 / 100 000
Denmark
Point prevalence
1-9 / 100 000
Denmark
Prevalence at birth
1-9 / 100 000
Sweden
Point prevalence
1-9 / 100 000
Sweden
Prevalence at birth
1-9 / 100 000
United Kingdom
Prevalence at birth
1-9 / 100 000
United States
Point prevalence
1-9 / 100 000
United States
Prevalence at birth
1-9 / 100 000
Europe
Point prevalence
1-9 / 100 000
Europe
Prevalence at birth
1-9 / 100 000
Netherlands
Prevalence at birth
1-5 / 10 000
Australia

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы