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Neonatal Marfan syndrome

ORPHA:284979DiseaseAutosomal dominantInfancy, Neonatal

Ассоциированные гены (1)

FBN1
fibrillin 1
Disease-causing germline mutation(s) in
OMIM: 134797

Фенотипы (43)

Облигатный (100%)3
HP:0001653Mitral regurgitation
HP:0002097Emphysema
HP:0005180Tricuspid regurgitation
Очень частый (80–99%)27
HP:0000268Dolichocephaly
HP:0000485Megalocornea
HP:0000768Pectus carinatum
HP:0000973Cutis laxa
HP:0001083Ectopia lentis
HP:0001166Arachnodactyly
HP:0001181Adducted thumb
HP:0001270Motor delay
HP:0001371Flexion contracture
HP:0001518Small for gestational age
HP:0001626Abnormality of the cardiovascular system
HP:0001634Mitral valve prolapse
HP:0001704Tricuspid valve prolapse
HP:0001713Abnormal cardiac ventricle morphology
HP:0002643Neonatal respiratory distress
HP:0004970Ascending tubular aorta aneurysm
HP:0008124Talipes calcaneovarus
HP:0008734Decreased testicular size
HP:0010511Long toe
HP:0011003High myopia
HP:0011968Feeding difficulties
HP:0012418Hypoxemia
HP:0030148Heart murmur
HP:0100578Lipoatrophy
HP:0100625Enlarged thorax
HP:0100693Iridodonesis
HP:0100807Long fingers
Частый (30–79%)13
HP:0000347Micrognathia
HP:0000369Low-set ears
HP:0000431Wide nasal bridge
HP:0000490Deeply set eye
HP:0000494Downslanted palpebral fissures
HP:0000592Blue sclerae
HP:0001252Hypotonia
HP:0001265Hyporeflexia
HP:0001382Joint hypermobility
HP:0002616Aortic root aneurysm
HP:0002705High, narrow palate
HP:0009901Crumpled ear
HP:0012771Increased arm span

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