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Mutilating palmoplantar keratoderma with periorificial keratotic plaques

ORPHA:659DiseaseAutosomal dominant, Not applicable, X-linked recessiveChildhood, Infancy, Neonatal

Ассоциированные гены (3)

PERP
p53 apoptosis effector related to PMP22
Disease-causing germline mutation(s) in
OMIM: 609301
MBTPS2
membrane bound transcription factor peptidase, site 2
Disease-causing germline mutation(s) in
OMIM: 300294
TRPV3
transient receptor potential cation channel subfamily V member 3
Disease-causing germline mutation(s) (gain of function) in
OMIM: 607066

Фенотипы (23)

Очень частый (80–99%)9
HP:0000970Anhidrosis
HP:0000982Palmoplantar keratoderma
HP:0001072Thickened skin
HP:0001231Abnormal fingernail morphology
HP:0007410Palmoplantar hyperhidrosis
HP:0008070Sparse hair
HP:0010783Erythema
HP:0031013Ankylosis
HP:0031057Skin fissure
Частый (30–79%)5
HP:0000164Abnormality of the dentition
HP:0000407Sensorineural hearing impairment
HP:0000668Hypodontia
HP:0000670Carious teeth
HP:0200042Skin ulcer
Периодический (5–29%)9
HP:0001596Alopecia
HP:0002797Osteolysis
HP:0002861Melanoma
HP:0008069Neoplasm of the skin
HP:0011830Abnormal oral mucosa morphology
HP:0100526Neoplasm of the lung
HP:0000157Abnormality of the tongue
HP:0000168Abnormality of the gingiva
HP:0001250Seizure

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