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Pyle disease

ORPHA:3005DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

SFRP4
secreted frizzled related protein 4
Disease-causing germline mutation(s) (loss of function) in
OMIM: 606570

Фенотипы (18)

Очень частый (80–99%)2
HP:0002857Genu valgum
HP:0004975Erlenmeyer flask deformity of the femurs
Частый (30–79%)8
HP:0000303Mandibular prognathia
HP:0000411Protruding ear
HP:0000670Carious teeth
HP:0000689Dental malocclusion
HP:0002659Increased susceptibility to fractures
HP:0003051Enlarged metaphyses
HP:0005464Craniofacial osteosclerosis
HP:0006599Medial widening of clavicles
Периодический (5–29%)8
HP:0000696Delayed eruption of permanent teeth
HP:0000926Platyspondyly
HP:0001324Muscle weakness
HP:0002645Wormian bones
HP:0002650Scoliosis
HP:0002829Arthralgia
HP:0003418Back pain
HP:0010539Thin calvarium

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