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Pachyonychia congenita

ORPHA:2309DiseaseAutosomal dominant, Autosomal recessiveAll ages

Ассоциированные гены (4)

KRT16
keratin 16
Disease-causing germline mutation(s) in
OMIM: 148067
KRT17
keratin 17
Disease-causing germline mutation(s) in
OMIM: 148069
KRT6A
keratin 6A
Disease-causing germline mutation(s) in
OMIM: 148041
KRT6B
keratin 6B
Disease-causing germline mutation(s) in
OMIM: 148042

Фенотипы (27)

Очень частый (80–99%)6
HP:0000982Palmoplantar keratoderma
HP:0007446Palmoplantar blistering
HP:0008401Onychogryposis of toenails
HP:0008404Nail dystrophy
HP:0012514Lower limb pain
HP:0030268Hyperplastic callus formation
Частый (30–79%)10
HP:0002745Oral leukoplakia
HP:0007410Palmoplantar hyperhidrosis
HP:0007490Linear arrays of macular hyperkeratoses in flexural areas
HP:0007502Follicular hyperkeratosis
HP:0010765Palmar hyperkeratosis
HP:0012035Steatocystoma multiplex
HP:0025245Cutaneous cyst
HP:0040036Onychogryposis of fingernail
HP:0100798Fingernail dysplasia
HP:0200040Epidermoid cyst
Периодический (5–29%)7
HP:0000695Natal tooth
HP:0001508Failure to thrive
HP:0001818Paronychia
HP:0006288Advanced eruption of teeth
HP:0011968Feeding difficulties
HP:0025248Eruptive vellus hair cyst
HP:0030766Ear pain
Очень редкий (1–4%)4
HP:0001596Alopecia
HP:0001609Hoarse voice
HP:0002098Respiratory distress
HP:0030318Angular cheilitis

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