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Combined immunodeficiency-multiple intestinal atresia

ORPHA:436252DiseaseAutosomal recessiveNeonatal

Ассоциированные гены (2)

TTC7A
tetratricopeptide repeat domain 7A
Disease-causing germline mutation(s) (loss of function) in
OMIM: 609332
PI4KA
phosphatidylinositol 4-kinase alpha
Disease-causing germline mutation(s) in
OMIM: 600286

Фенотипы (30)

Очень частый (80–99%)4
HP:0002589Gastrointestinal atresia
HP:0004430Severe combined immunodeficiency
HP:0010766Ectopic calcification
HP:0011100Intestinal atresia
Частый (30–79%)8
HP:0001511Intrauterine growth retardation
HP:0001561Polyhydramnios
HP:0002223Absent eyebrow
HP:0002721Immunodeficiency
HP:0003270Abdominal distention
HP:0005229Jejunoileal ulceration
HP:0008070Sparse hair
HP:0025085Bloody diarrhea
Периодический (5–29%)8
HP:0000778Hypoplasia of the thymus
HP:0001072Thickened skin
HP:0002293Alopecia of scalp
HP:0002566Intestinal malrotation
HP:0002722Recurrent abscess formation
HP:0005224Rectal abscess
HP:0100592Peritoneal abscess
HP:0100889Abnormality of the ductus choledochus
Очень редкий (1–4%)10
HP:0000872Hashimoto thyroiditis
HP:0001539Omphalocele
HP:0001629Ventricular septal defect
HP:0001890Autoimmune hemolytic anemia
HP:0002960Autoimmunity
HP:0003765Psoriasiform dermatitis
HP:0008404Nail dystrophy
HP:0010959Congenital pulmonary airway malformation
HP:0012115Hepatitis
HP:0100651Type I diabetes mellitus

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы