Tezacaftor, Ivacaftor Combination Generally Safe, Well Tolerated in Children With CF Under Age 12
Data from a phase 3 trial are supporting the use of tezacaftor in combination with ivacaftor for the treatment of cystic fibrosis (CF) in...
Different CFTR Potentiators Bind to Same Flexible Spot on Protein, Study Finds
Approved and investigative cystic fibrosis (CF) treatments known as CFTR potentiators work by binding to the same flexible protein spot —...
Kalydeco’s Safety and Benefits Studied in CF Patients with Severe Lung Disease
Treatment with Kalydeco (ivacaftor) improved lung function, exercise capacity, and sweat chloride concentration in cystic fibrosis (CF)...
FDA Approves Symdeko for Treatment of CF Children, Ages 6 to 11, with Certain CFTR Mutations
The U.S. Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor) tablets for the treatment of...
FDA Approves Kalydeco for Treatment of 6- to 12-month-old Infants with Cystic Fibrosis
The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for the treatment of infants from six to 12 months old, who...
Cystic fibrosis drug combo found safe and effective for 2–5 year olds
An open label phase 3 study conducted at Ann & Robert H. Lurie Children’s Hospital of Chicago and other centers established that a...
Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests
The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically...
Triple Combo Treatment Improves Lung Function in Patients with Cystic Fibrosis
An investigational triple combination treatment regimen for cystic fibrosis (CF) improved patients’ lung function in 2 phase 3 studies,...
New cystic fibrosis drugs significantly improve lung function
A triple drug combination for treating the underlying cause of cystic fibrosis is currently under study. The preliminary results show a...
The Bittersweet Downside of CFTR Modulators
I’ve been on Orkambi (lumacaftor/ivacaftor), a cystic fibrosis transmembrane conductance regulator (CFTR) modulator, for just over three...
