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Fragile X syndrome

ORPHA:908Malformation syndromeX-linked dominantChildhood, Infancy, Neonatal

Ассоциированные гены (1)

FMR1
fragile X messenger ribonucleoprotein 1
Disease-causing germline mutation(s) (loss of function) in
OMIM: 309550

Фенотипы (33)

Очень частый (80–99%)7
HP:0001382Joint hypermobility
HP:0000053Macroorchidism
HP:0000389Chronic otitis media
HP:0001763Pes planus
HP:0002167Abnormality of speech or vocalization
HP:0002342Intellectual disability, moderate
HP:0003564Folate-dependent fragile site at Xq28
Частый (30–79%)17
HP:0000246Sinusitis
HP:0000256Macrocephaly
HP:0000275Narrow face
HP:0000276Long face
HP:0000303Mandibular prognathia
HP:0000388Otitis media
HP:0000400Macrotia
HP:0000411Protruding ear
HP:0000750Delayed speech and language development
HP:0001252Hypotonia
HP:0002003Large forehead
HP:0002007Frontal bossing
HP:0002020Gastroesophageal reflux
HP:0002194Delayed gross motor development
HP:0002360Sleep abnormality
HP:0002650Scoliosis
HP:0007018Attention deficit hyperactivity disorder
Периодический (5–29%)9
HP:0000486Strabismus
HP:0000717Autism
HP:0000737Irritability
HP:0000739Anxiety
HP:0001250Seizure
HP:0001634Mitral valve prolapse
HP:0002120Cerebral cortical atrophy
HP:0004970Ascending tubular aorta aneurysm
HP:0100716Self-injurious behavior

Эпидемиология (7)

Point prevalence
1-5 / 10 000
Worldwide
Point prevalence
1-5 / 10 000
Europe
Prevalence at birth
1-9 / 100 000
United States
Prevalence at birth
1-9 / 100 000
France
Prevalence at birth
1-5 / 10 000
Spain
Prevalence at birth
1-5 / 10 000
Canada
Prevalence at birth
1-9 / 100 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы