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Acromesomelic dysplasia, Maroteaux type

ORPHA:40Malformation syndromeAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

NPR2
natriuretic peptide receptor 2
Disease-causing germline mutation(s) in
OMIM: 108961

Фенотипы (18)

Частый (30–79%)18
HP:0001382Joint hypermobility
HP:0000268Dolichocephaly
HP:0000912Sprengel anomaly
HP:0001156Brachydactyly
HP:0001387Joint stiffness
HP:0002007Frontal bossing
HP:0002650Scoliosis
HP:0002808Kyphosis
HP:0003086Acromesomelia
HP:0003300Ovoid vertebral bodies
HP:0003307Hyperlordosis
HP:0003312Abnormal form of the vertebral bodies
HP:0003498Disproportionate short stature
HP:0004568Beaking of vertebral bodies
HP:0005280Depressed nasal bridge
HP:0006487Bowing of the long bones
HP:0008422Vertebral wedging
HP:0011220Prominent forehead

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