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3M syndrome

ORPHA:2616Malformation syndromeAutosomal recessiveAntenatal

Ассоциированные гены (3)

CUL7
cullin 7
Disease-causing germline mutation(s) in
OMIM: 609577
OBSL1
obscurin like cytoskeletal adaptor 1
Disease-causing germline mutation(s) in
OMIM: 610991
CCDC8
coiled-coil domain containing 8 subunit of 3M complex
Disease-causing germline mutation(s) in
OMIM: 614145

Фенотипы (42)

Очень частый (80–99%)20
HP:0000232Everted lower lip vermilion
HP:0000325Triangular face
HP:0000337Broad forehead
HP:0000414Bulbous nose
HP:0000463Anteverted nares
HP:0000470Short neck
HP:0000574Thick eyebrow
HP:0000944Abnormal metaphysis morphology
HP:0001511Intrauterine growth retardation
HP:0001838Rocker bottom foot
HP:0002007Frontal bossing
HP:0002750Delayed skeletal maturation
HP:0003100Slender long bone
HP:0003173Hypoplastic pubic bone
HP:0003175Hypoplastic ischia
HP:0003691Scapular winging
HP:0004322Short stature
HP:0004570Increased vertebral height
HP:0008839Hypoplastic pelvis
HP:0011800Midface retrusion
Частый (30–79%)15
HP:0000268Dolichocephaly
HP:0000307Pointed chin
HP:0000343Long philtrum
HP:0000411Protruding ear
HP:0000682Abnormality of dental enamel
HP:0000684Delayed eruption of teeth
HP:0000883Thin ribs
HP:0000888Horizontal ribs
HP:0002983Micromelia
HP:0003022Hypoplasia of the ulna
HP:0003307Hyperlordosis
HP:0009811Abnormality of the elbow
HP:0010306Short thorax
HP:0100625Enlarged thorax
HP:0001382Joint hypermobility
Периодический (5–29%)7
HP:0000047Hypospadias
HP:0000144Decreased fertility
HP:0001374Congenital hip dislocation
HP:0002650Scoliosis
HP:0002808Kyphosis
HP:0004209Clinodactyly of the 5th finger
HP:0100659Abnormality of the cerebral vasculature

Эпидемиология (2)

Cases/families
Worldwide
Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы