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Neurofibromatosis-Noonan syndrome

ORPHA:638Malformation syndromeAutosomal dominantInfancy, Neonatal

Ассоциированные гены (2)

MAP2K2
mitogen-activated protein kinase kinase 2
Disease-causing germline mutation(s) in
OMIM: 601263
NF1
neurofibromin 1
Disease-causing germline mutation(s) in
OMIM: 613113

Фенотипы (18)

Очень частый (80–99%)13
HP:0000271Abnormality of the face
HP:0000316Hypertelorism
HP:0000465Webbed neck
HP:0000494Downslanted palpebral fissures
HP:0000508Ptosis
HP:0001328Specific learning disability
HP:0001639Hypertrophic cardiomyopathy
HP:0001642Pulmonic stenosis
HP:0004322Short stature
HP:0007565Multiple cafe-au-lait spots
HP:0009023Abdominal wall muscle weakness
HP:0011039Abnormality of the helix
HP:0000358Posteriorly rotated ears
Частый (30–79%)5
HP:0000028Cryptorchidism
HP:0000765Abnormal thorax morphology
HP:0002015Dysphagia
HP:0003010Prolonged bleeding time
HP:0100763Abnormality of the lymphatic system

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы