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RHYNS syndrome

ORPHA:140976DiseaseAutosomal recessiveAntenatal, Infancy, Neonatal

Ассоциированные гены (1)

TMEM67
transmembrane protein 67
Disease-causing germline mutation(s) in
OMIM: 609884

Фенотипы (19)

Очень частый (80–99%)6
HP:0000090Nephronophthisis
HP:0000508Ptosis
HP:0000510Rod-cone dystrophy
HP:0000924Abnormality of the skeletal system
HP:0001392Abnormality of the liver
HP:0040075Hypopituitarism
Частый (30–79%)13
HP:0000002Abnormality of body height
HP:0000003Multicystic kidney dysplasia
HP:0000365Hearing impairment
HP:0000490Deeply set eye
HP:0000938Osteopenia
HP:0000946Hypoplastic ilia
HP:0002652Skeletal dysplasia
HP:0002750Delayed skeletal maturation
HP:0003170Abnormality of the acetabulum
HP:0006824Cranial nerve paralysis
HP:0006897Abducens palsy
HP:0010585Small epiphyses
HP:0011314Abnormality of long bone morphology

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