Orkambi – Lumacaftor / Ivakaftor uses, dose and side effects
Orkambi contains two active substances, luma kaftar and ivakaftar. It is a drug used for the long-term treatment of cystic fibrosis (CF) in patients aged 6 years and older with a specific change (called F508del – mutation ) in the gene for a protein called CFTR (which stands for Cystic Fibrosis Transmembrane Conductance Regulator) and is important for the regulation of mucus in the lungs. In people with mutation one, the CFTR protein is formed abnormally. The cells contain two copies of the CFTR gene. Orkambi is used in patients in whom both copies have F508del – mutation one (homozygous is).
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