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Severe combined immunodeficiency due to adenosine deaminase deficiency

ORPHA:277DiseaseAutosomal recessiveInfancy, Neonatal

Ассоциированные гены (1)

ADA
adenosine deaminase
Disease-causing germline mutation(s) in
OMIM: 608958

Фенотипы (20)

Частый (30–79%)20
HP:0000246Sinusitis
HP:0000403Recurrent otitis media
HP:0001508Failure to thrive
HP:0001888Lymphopenia
HP:0002014Diarrhea
HP:0002788Recurrent upper respiratory tract infections
HP:0002849Absence of lymph node germinal center
HP:0002960Autoimmunity
HP:0003212Increased circulating IgE level
HP:0005354Lack of T cell function
HP:0005368Abnormality of humoral immunity
HP:0005390Recurrent opportunistic infections
HP:0005403Decreased total T cell count
HP:0006532Recurrent pneumonia
HP:0010444Pulmonary insufficiency
HP:0010976Decreased total B cell count
HP:0011123Inflammatory abnormality of the skin
HP:0012393Allergy
HP:0025379Anti-thyroid peroxidase antibody positivity
HP:0030813Absent tonsils

Эпидемиология (2)

Prevalence at birth
1-9 / 1 000 000
Europe
Point prevalence
1-9 / 1 000 000
Europe

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы