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Fetal akinesia deformation sequence

ORPHA:994Malformation syndromeAutosomal recessiveAntenatal, Neonatal

Ассоциированные гены (10)

SLC18A3
solute carrier family 18 member A3
Disease-causing germline mutation(s) in
OMIM: 600336
MYOD1
myogenic differentiation 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 159970
RAPSN
receptor associated protein of the synapse
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601592
DOK7
docking protein 7
Disease-causing germline mutation(s) (loss of function) in
OMIM: 610285
MUSK
muscle associated receptor tyrosine kinase
Disease-causing germline mutation(s) (loss of function) in
OMIM: 601296
NUP88
nucleoporin 88
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602552
GLDN
gliomedin
Disease-causing germline mutation(s) in
OMIM: 608603
KIF21A
kinesin family member 21A
Disease-causing germline mutation(s) (loss of function) in
OMIM: 608283
MAGEL2
MAGE family member L2
Disease-causing germline mutation(s) in
OMIM: 605283
TUBA1A
tubulin alpha 1a
Disease-causing germline mutation(s) in
OMIM: 602529

Фенотипы (24)

Очень частый (80–99%)12
HP:0000347Micrognathia
HP:0001262Excessive daytime somnolence
HP:0001511Intrauterine growth retardation
HP:0001989Fetal akinesia sequence
HP:0002089Pulmonary hypoplasia
HP:0002093Respiratory insufficiency
HP:0002304Akinesia
HP:0002375Hypokinesia
HP:0002804Arthrogryposis multiplex congenita
HP:0002828Multiple joint contractures
HP:0010489Absent palmar crease
HP:0100490Camptodactyly of finger
Частый (30–79%)9
HP:0000028Cryptorchidism
HP:0000175Cleft palate
HP:0000316Hypertelorism
HP:0000358Posteriorly rotated ears
HP:0000476Cystic hygroma
HP:0001561Polyhydramnios
HP:0002650Scoliosis
HP:0003700Generalized amyotrophy
HP:0005280Depressed nasal bridge
Периодический (5–29%)3
HP:0001059Pterygium
HP:0001305Dandy-Walker malformation
HP:0005245Intestinal hypoplasia

Эпидемиология (2)

Prevalence at birth
1-9 / 1 000 000
Europe
Point prevalence
<1 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы