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Melnick-Needles syndrome

ORPHA:2484Malformation syndromeX-linked dominantChildhood

Ассоциированные гены (1)

FLNA
filamin A
Disease-causing germline mutation(s) (gain of function) in
OMIM: 300017

Фенотипы (35)

Очень частый (80–99%)11
HP:0000270Delayed cranial suture closure
HP:0000316Hypertelorism
HP:0000336Prominent supraorbital ridges
HP:0000347Micrognathia
HP:0000520Proptosis
HP:0000774Narrow chest
HP:0000944Abnormal metaphysis morphology
HP:0003103Abnormal cortical bone morphology
HP:0004322Short stature
HP:0006487Bowing of the long bones
HP:0010306Short thorax
Частый (30–79%)22
HP:0001382Joint hypermobility
HP:0000076Vesicoureteral reflux
HP:0000126Hydronephrosis
HP:0000293Full cheeks
HP:0000324Facial asymmetry
HP:0000365Hearing impairment
HP:0000684Delayed eruption of teeth
HP:0000692Tooth malposition
HP:0000772Abnormal rib morphology
HP:0000894Short clavicles
HP:0001671Abnormal cardiac septum morphology
HP:0002007Frontal bossing
HP:0002205Recurrent respiratory infections
HP:0002650Scoliosis
HP:0002673Coxa valga
HP:0002827Hip dislocation
HP:0002879Anisospondyly
HP:0003172Abnormality of the pubic bone
HP:0004493Craniofacial hyperostosis
HP:0009771Osteolytic defects of the phalanges of the hand
HP:0009882Short distal phalanx of finger
HP:0010230Cone-shaped epiphyses of the phalanges of the hand
Периодический (5–29%)2
HP:0001539Omphalocele
HP:0002093Respiratory insufficiency

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