A new drug called "Lynovex" just got approved by FDA that has unique multi-functional, combined properties of breaking down the excessive mucus produced in the airways. The new drug allows for destruction of the bacteria responsible for the recurrent respiratory infections in CF by disrupting/preventing the biofilms, which these bacteria typically form. By functioning in this way, it could improve lung function and halt or prevent long term damage or degeneration of respiratory
It is so crucial that insurers cover drugs like Kalydeco and here is another milestone for the Kalydeco drug and the CF community. We must educate the payers of healthcare and make them understand that even though Kalydeco costs are high, they offset an enormous treatment cost burden for them as well as providing a bright and productive future to teenagers like 13 year old Larissa Rueckenbach.
If there's one thing I have learned about CFers, it is that they are the most incredible and
Updated Yesterday at 05:19 AM by ChrisBaldwin
My memoir (getting to 40 with CF) called 'How have I cheated death?' has been nominated for the People's Book Prize! Please help me to win before the end of November 2014 by voting for me via this link.
Thanks very much!
ProQR IPO is this week. Their leading product is QR-010 which aims to restore the defect in CFTR protein caused by the del F508 mutation. Specifically, administration into the lungs of a chemically modified oligoribonucleotide that is capable of restoring the RNA sequence coding for CFTR, thereby restoring CFTR protein function, or at least improving it. Administration would be via a nebulized drug+hypertonic saline solution. Cost is not yet known to me. IPO is for the rollout of phase 1 clinical
Updated 09-16-2014 at 04:34 AM by ChrisBaldwin
THANK YOU! We gave an amazing presentation this morning at the ExL ePatient Connections Conference in Philadelphia and we have this wonderful CF community to thank for driving and motivating us to present. We presented a topic on "participatory healthcare" and why industry should care about the CF community and how they can engage with us in order to improve patient outcomes. Its been an honor to represent you and we can honestly say this presentation is a springboard to some incredible
Updated Yesterday at 05:17 AM by ChrisBaldwin