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Papillon-Lefèvre syndrome

ORPHA:678DiseaseAutosomal recessiveChildhood, Infancy, Neonatal

Ассоциированные гены (1)

CTSC
cathepsin C
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602365

Фенотипы (29)

Очень частый (80–99%)11
HP:0000164Abnormality of the dentition
HP:0000166Severe periodontitis
HP:0000230Gingivitis
HP:0000704Periodontitis
HP:0000972Palmoplantar hyperkeratosis
HP:0000982Palmoplantar keratoderma
HP:0001231Abnormal fingernail morphology
HP:0006308Atrophy of alveolar ridges
HP:0006323Premature loss of primary teeth
HP:0009804Tooth agenesis
HP:0200039Pustule
Частый (30–79%)7
HP:0001581Recurrent skin infections
HP:0001597Abnormality of the nail
HP:0002205Recurrent respiratory infections
HP:0002514Cerebral calcification
HP:0008404Nail dystrophy
HP:0011132Chronic furunculosis
HP:0100838Recurrent cutaneous abscess formation
Периодический (5–29%)11
HP:0000998Hypertrichosis
HP:0001053Hypopigmented skin patches
HP:0001073Cigarette-paper scars
HP:0001166Arachnodactyly
HP:0002230Generalized hirsutism
HP:0002231Sparse body hair
HP:0002797Osteolysis
HP:0002860Squamous cell carcinoma
HP:0002861Melanoma
HP:0008069Neoplasm of the skin
HP:0100523Liver abscess

Эпидемиология (1)

Point prevalence
1-9 / 1 000 000
Worldwide

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы