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Freeman-Sheldon syndrome

ORPHA:2053Malformation syndromeAutosomal dominant, Autosomal recessiveAntenatal, Neonatal

Ассоциированные гены (2)

NALCN
sodium leak channel, non-selective
Disease-causing germline mutation(s) in
OMIM: 611549
MYH3
myosin heavy chain 3
Disease-causing germline mutation(s) in
OMIM: 160720

Фенотипы (31)

Очень частый (80–99%)16
HP:0000160Narrow mouth
HP:0000164Abnormality of the dentition
HP:0000316Hypertelorism
HP:0000430Underdeveloped nasal alae
HP:0000431Wide nasal bridge
HP:0000457Depressed nasal ridge
HP:0000494Downslanted palpebral fissures
HP:0001387Joint stiffness
HP:0001508Failure to thrive
HP:0001510Growth delay
HP:0001762Talipes equinovarus
HP:0002650Scoliosis
HP:0008872Feeding difficulties in infancy
HP:0009465Ulnar deviation of finger
HP:0010751Chin dimple
HP:0100490Camptodactyly of finger
Частый (30–79%)11
HP:0000028Cryptorchidism
HP:0000343Long philtrum
HP:0000365Hearing impairment
HP:0000486Strabismus
HP:0000490Deeply set eye
HP:0000508Ptosis
HP:0001557Prenatal movement abnormality
HP:0001611Hypernasal speech
HP:0002047Malignant hyperthermia
HP:0002167Abnormality of speech or vocalization
HP:0004322Short stature
Периодический (5–29%)4
HP:0001561Polyhydramnios
HP:0001562Oligohydramnios
HP:0010489Absent palmar crease
HP:0100790Hernia

Эпидемиология (2)

Point prevalence
<1 / 1 000 000
Worldwide
Cases/families
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы