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Laurence-Moon syndrome

ORPHA:2377Malformation syndromeAutosomal recessiveNeonatal

Ассоциированные гены (1)

PNPLA6
patatin like domain 6, lysophospholipase
Disease-causing germline mutation(s) (loss of function) in
OMIM: 603197

Фенотипы (24)

Очень частый (80–99%)5
HP:0001161Hand polydactyly
HP:0001249Intellectual disability
HP:0001513Obesity
HP:0006101Finger syndactyly
HP:0009896Abnormality of the antitragus
Частый (30–79%)5
HP:0000028Cryptorchidism
HP:0000083Renal insufficiency
HP:0000407Sensorineural hearing impairment
HP:0004322Short stature
HP:0008736Hypoplasia of penis
Периодический (5–29%)14
HP:0000248Brachycephaly
HP:0000286Epicanthus
HP:0000486Strabismus
HP:0000518Cataract
HP:0000612Iris coloboma
HP:0000639Nystagmus
HP:0001156Brachydactyly
HP:0001251Ataxia
HP:0002612Congenital hepatic fibrosis
HP:0005978Type II diabetes mellitus
HP:0007598Bilateral single transverse palmar creases
HP:0030680Abnormal cardiovascular system morphology
HP:0100627Displacement of the urethral meatus
HP:0000358Posteriorly rotated ears

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