Orphan diseases

ORPHAN
DISEASES

MIK LLC, as part of specialized research programs and non-interventional studies to study the prevalence and choice of etiopathogenetic therapy for some rare (orphan) diseases, provides assistance in solving the problems of diagnosing and treating patients.

Cystic fibrosis (CF)
Phenylketonuria (PKU)

CYSTIC FIBROSIS

Research program to collect data on the spectrum of mutations in the CFTR gene to predict the clinical course of the disease and the choice of etiopathogenetic therapy in patients with cystic fibrosis in the Russian Federation.

Drugs not registered in the Russian Federation

TRILEXA®
(Elexacaftor 100mg, Ivacaftor 75mg, Tezacaftor 50mg + Ivacaftor 150mg). The package contains 60 orange film-coated tablets containing the combination elexacaftor/tezacaftor/ivacaftor and 30 blue film-coated tablets containing ivacaftor.

INSTRUCTIONS FOR USE

TISURAL®
Levofloxacin, solution for inhalation 240 mg №56

INSTRUCTIONS FOR USE

Drugs registered in the Russian Federation

Tobramycin-Gobbi®
Tobramycin, solution for inhalation 60 mg/ml 5 ml №56

INSTRUCTIONS FOR USE PUBLICATIONS

PHENYLKETONURIA

Efkuria®
(Sapropterin), dispersible tablets 100 mg №30

INSTRUCTIONS FOR USE