← Назад

Cystic fibrosis

ORPHA:586DiseaseAutosomal recessiveAll ages

Ассоциированные гены (19)

SERPINA1
serpin family A member 1
Modifying germline mutation in
OMIM: 107400
SLC26A9
solute carrier family 26 member 9
Modifying germline mutation in
OMIM: 608481
SLC6A14
solute carrier family 6 member 14
Modifying germline mutation in
OMIM: 300444
SLC9A3
solute carrier family 9 member A3
Modifying germline mutation in
OMIM: 182307
CEACAM3
CEA cell adhesion molecule 3
Modifying germline mutation in
OMIM: 609142
CEACAM6
CEA cell adhesion molecule 6
Modifying germline mutation in
OMIM: 163980
EDNRA
endothelin receptor type A
Modifying germline mutation in
OMIM: 131243
GSTM3
glutathione S-transferase mu 3
Modifying germline mutation in
OMIM: 138390
HMOX1
heme oxygenase 1
Modifying germline mutation in
OMIM: 141250
GCLC
glutamate-cysteine ligase catalytic subunit
Modifying germline mutation in
OMIM: 606857
HFE
homeostatic iron regulator
Modifying germline mutation in
OMIM: 613609
CFTR
CF transmembrane conductance regulator
Disease-causing germline mutation(s) (loss of function) in
OMIM: 602421
TGFB1
transforming growth factor beta 1
Modifying germline mutation in
OMIM: 190180
DCTN4
dynactin subunit 4
Modifying germline mutation in
OMIM: 614758
CLCA4
chloride channel accessory 4
Modifying germline mutation in
OMIM: 616857
STX1A
syntaxin 1A
Modifying germline mutation in
OMIM: 186590
KCNN4
potassium calcium-activated channel subfamily N member 4
Modifying germline mutation in
OMIM: 602754
MIF
macrophage migration inhibitory factor
Modifying germline mutation in
OMIM: 153620
SLC11A1
solute carrier family 11 member 1
Modifying germline mutation in
OMIM: 600266

Фенотипы (35)

Очень частый (80–99%)7
HP:0001738Exocrine pancreatic insufficiency
HP:0002024Malabsorption
HP:0002110Bronchiectasis
HP:0002205Recurrent respiratory infections
HP:0006536Airway obstruction
HP:0012236Elevated sweat chloride
HP:0012873Absent vas deferens
Частый (30–79%)2
HP:0001508Failure to thrive
HP:0003251Male infertility
Периодический (5–29%)17
HP:0000716Depression
HP:0000739Anxiety
HP:0000938Osteopenia
HP:0001392Abnormality of the liver
HP:0002020Gastroesophageal reflux
HP:0002035Rectal prolapse
HP:0002099Asthma
HP:0002570Steatorrhea
HP:0002724Recurrent Aspergillus infections
HP:0002726Recurrent Staphylococcus aureus infections
HP:0002783Recurrent lower respiratory tract infections
HP:0002910Elevated circulating hepatic transaminase concentration
HP:0004401Meconium ileus
HP:0005376Recurrent Haemophilus influenzae infections
HP:0032342Reduced forced expiratory volume in one second
HP:0045082Decreased body mass index
HP:0000246Sinusitis
Очень редкий (1–4%)9
HP:0000365Hearing impairment
HP:0000787Nephrolithiasis
HP:0000939Osteoporosis
HP:0001394Cirrhosis
HP:0002105Hemoptysis
HP:0002107Pneumothorax
HP:0002842Recurrent Burkholderia cepacia infections
HP:0032261Nontuberculous mycobacterial pulmonary infection
HP:0100582Nasal polyposis

Эпидемиология (62)

Prevalence at birth
1-5 / 10 000
Europe
Point prevalence
1-5 / 10 000
Europe
Prevalence at birth
1-5 / 10 000
Austria
Point prevalence
1-9 / 100 000
Austria
Prevalence at birth
1-5 / 10 000
Belgium
Point prevalence
1-5 / 10 000
Belgium
Prevalence at birth
1-5 / 10 000
Bulgaria
Point prevalence
1-9 / 100 000
Bulgaria
Prevalence at birth
1-5 / 10 000
Cyprus
Point prevalence
1-9 / 100 000
Cyprus
Prevalence at birth
1-5 / 10 000
Czech Republic
Point prevalence
1-9 / 100 000
Czech Republic
Prevalence at birth
1-5 / 10 000
Denmark
Point prevalence
1-9 / 100 000
Denmark
Prevalence at birth
1-5 / 10 000
Estonia
Point prevalence
1-9 / 100 000
Estonia
Prevalence at birth
1-9 / 100 000
Finland
Point prevalence
1-9 / 100 000
Finland
Prevalence at birth
1-5 / 10 000
France
Point prevalence
1-5 / 10 000
France
Prevalence at birth
1-5 / 10 000
Germany
Point prevalence
1-5 / 10 000
Germany
Prevalence at birth
1-5 / 10 000
Greece
Point prevalence
1-9 / 100 000
Greece
Prevalence at birth
Unknown
Hungary
Point prevalence
1-9 / 100 000
Hungary
Prevalence at birth
1-5 / 10 000
Ireland
Point prevalence
1-5 / 10 000
Ireland
Prevalence at birth
1-5 / 10 000
Italy
Point prevalence
1-5 / 10 000
Italy
Prevalence at birth
Unknown
Latvia
Point prevalence
1-9 / 100 000
Latvia
Prevalence at birth
Unknown
Lithuania
Point prevalence
1-9 / 100 000
Lithuania
Prevalence at birth
Unknown
Luxembourg
Point prevalence
1-9 / 100 000
Luxembourg
Prevalence at birth
Unknown
Malta
Point prevalence
1-9 / 100 000
Malta
Prevalence at birth
1-5 / 10 000
Netherlands
Point prevalence
1-9 / 100 000
Netherlands
Prevalence at birth
1-5 / 10 000
Norway
Point prevalence
1-9 / 100 000
Norway
Prevalence at birth
1-5 / 10 000
Poland
Point prevalence
1-5 / 10 000
Poland
Prevalence at birth
1-5 / 10 000
Portugal
Point prevalence
1-9 / 100 000
Portugal
Prevalence at birth
1-5 / 10 000
Romania
Point prevalence
1-9 / 100 000
Romania
Prevalence at birth
1-9 / 100 000
Russian Federation
Point prevalence
1-9 / 100 000
Russian Federation
Prevalence at birth
1-5 / 10 000
Slovakia
Point prevalence
1-9 / 100 000
Slovakia
Prevalence at birth
1-5 / 10 000
Slovenia
Point prevalence
1-9 / 100 000
Slovenia
Prevalence at birth
1-5 / 10 000
Spain
Point prevalence
1-9 / 100 000
Spain
Prevalence at birth
1-5 / 10 000
Sweden
Point prevalence
1-5 / 10 000
Sweden
Prevalence at birth
1-5 / 10 000
United Kingdom
Point prevalence
1-5 / 10 000
United Kingdom
Annual incidence
1-5 / 10 000
Brazil
Point prevalence
1-9 / 1 000 000
China

Лекарства и терапия

Источник: Open Targets Platform (в реальном времени)

Клинические исследования

Источник: ClinicalTrials.gov (в реальном времени)

Внешние ресурсы