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Lujan-Fryns syndrome

ORPHA:776Malformation syndromeX-linked recessiveInfancy, Neonatal

Ассоциированные гены (3)

MED12
mediator complex subunit 12
Disease-causing germline mutation(s) in
OMIM: 300188
ZDHHC9
zDHHC palmitoyltransferase 9
Disease-causing germline mutation(s) in
OMIM: 300646
UPF3B
UPF3B regulator of nonsense mediated mRNA decay
Disease-causing germline mutation(s) in
OMIM: 300298

Фенотипы (34)

Очень частый (80–99%)12
HP:0000218High palate
HP:0000256Macrocephaly
HP:0000347Micrognathia
HP:0000348High forehead
HP:0000708Atypical behavior
HP:0001249Intellectual disability
HP:0001252Hypotonia
HP:0001519Disproportionate tall stature
HP:0001608Abnormality of the voice
HP:0001611Hypernasal speech
HP:0002167Abnormality of speech or vocalization
HP:0002650Scoliosis
Частый (30–79%)11
HP:0000053Macroorchidism
HP:0000275Narrow face
HP:0000322Short philtrum
HP:0000327Hypoplasia of the maxilla
HP:0000426Prominent nasal bridge
HP:0000767Pectus excavatum
HP:0001166Arachnodactyly
HP:0001631Atrial septal defect
HP:0007018Attention deficit hyperactivity disorder
HP:0007370Aplasia/Hypoplasia of the corpus callosum
HP:0001382Joint hypermobility
Периодический (5–29%)11
HP:0000164Abnormality of the dentition
HP:0000248Brachycephaly
HP:0000369Low-set ears
HP:0000411Protruding ear
HP:0000678Dental crowding
HP:0000709Psychosis
HP:0000738Hallucinations
HP:0001156Brachydactyly
HP:0001250Seizure
HP:0100490Camptodactyly of finger
HP:0100753Schizophrenia

Эпидемиология (1)

Point prevalence
Unknown
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы