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Borjeson-Forssman-Lehmann syndrome

ORPHA:127Malformation syndromeX-linked recessiveNeonatal

Ассоциированные гены (1)

PHF6
PHD finger protein 6
Disease-causing germline mutation(s) (loss of function) in
OMIM: 300414

Фенотипы (34)

Очень частый (80–99%)16
HP:0000028Cryptorchidism
HP:0000046Small scrotum
HP:0000135Hypogonadism
HP:0000280Coarse facial features
HP:0000771Gynecomastia
HP:0001182Tapered finger
HP:0001249Intellectual disability
HP:0001252Hypotonia
HP:0001769Broad foot
HP:0001831Short toe
HP:0001836Camptodactyly of toe
HP:0001956Truncal obesity
HP:0008070Sparse hair
HP:0008734Decreased testicular size
HP:0008736Hypoplasia of penis
HP:0009748Large earlobe
Частый (30–79%)6
HP:0000336Prominent supraorbital ridges
HP:0000490Deeply set eye
HP:0000508Ptosis
HP:0000574Thick eyebrow
HP:0000581Blepharophimosis
HP:0008872Feeding difficulties in infancy
Периодический (5–29%)12
HP:0001382Joint hypermobility
HP:0000202Orofacial cleft
HP:0000252Microcephaly
HP:0000256Macrocephaly
HP:0000365Hearing impairment
HP:0000518Cataract
HP:0000639Nystagmus
HP:0001250Seizure
HP:0003202Skeletal muscle atrophy
HP:0003272Abnormality of the hip bone
HP:0004322Short stature
HP:0009830Peripheral neuropathy

Эпидемиология (2)

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

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы