Cystic Fibrosis News - Week 35:
Researchers develop new lung-on-chip model for laboratory studies of respiratory challenges - http://t.co/zW3ZrQgTeq
Very interesting article on specialty drugs of which Kalydeco is a member - 1% of RX but 25% of spending! - http://t.co/ZEp9tSfxt7
CFers need...
A fantastic TED talk on "Participatory Healthcare" and what it can mean for the patient. Professor Bas Bloem has done some amazing things in the Netherlands on Parkinson's and his participatory healthcare model would be so valuable for the CF community! I will be speaking with Professor Bas...
A BREAKTHROUGH innovation just hit the market, which allows for the first time, to measure CF treatments in the lungs in "real-time". The new technique allows for doctors and researchers to measure how effective treatments are "very quickly", meaning they can adopt new treatments in clinical...
A SIGNIFICANT BREAKTHROUGH has occurred from scientist studying pigs tracheas that have been modified to mimic CF lungs. They discovered that CF lungs do not produce a very important and almost undetectable thin layer of fluid that lines the lungs, called "airway surface liquid" (ASL for short)...
A quick (easy to read) blurb on the recent drug combination therapies involving Kalydeco: The Kalydeco drug that effectively restores the flow of water and chloride through the CFTR protein channel has been successfully used to treat people with CF who carry the "celtic gene" (G551D), which...
It really comes down to how you quantify CFTR function... depending on the measurement and technique used very different conclusions can be drawn in most cases. And the study I refer is specific to CFTR function in the lungs. This gene therapy also has a local effect only in the lungs...so you...
Hi Aboveallislove, we are both correct and referencing sound studies. I am a scientist and I can tell you that we are both correct and basically comparing apples and oranges :)
What the video above of Prof. Eric Alton who explains CFTR function that "maybe" needed for sufficient lung function...
Cystic Fibrosis News - Week 34:
Dutch CF biotech ProQR Therapeutics files for a $75 million IPO [read]
Breakthrough: real-time tracking of treatments for cystic fibrosis. [read]
Telemedicine and the US Congress Why CARE? [read]
CF Trust demands a fair travel deal as insurance and airline...
A BREAKTHROUGH has occurred in the past week that will allow for real-time tracking of treatments for cystic fibrosis:
“At the moment we typically need to wait for a cystic fibrosis treatment to have an effect on lung health, measured by either a lung CT scan or breath measurement, to see...
Why combination therapies to treat the underlying cause of Cystic Fibrosis are a promising strategy?
There is great promise on the horizon for new combination therapies that treat the underlying cause of Cystic Fibrosis. Cystic Fibrosis is a result of specific mutations that occurs in the...
There is a big difference between CFTR function and lung function. What you are refering to is lung function. I am referring to CFTR function. Lung function is an outcome of many complex factors of course....CFTR obviously has an important role to play in the function for people with CF.
I am...
It is very possible (and likely) that a drug targeted to RNA would have an additive effect to the protein corrector/potentiator combos from Vertex. If this turns out to be the case it would be very exciting and may indeed result in greater restoration of organ function (lungs being the most...
Dutch CF biotech ProQR Therapeutics files for a $75 million IPO
If their claims turn out to be true, this could very well lead to a new drug for CF that would be suitable for a large % of CFers. It would also give Vertex some "healthy" competition, which is what we need in this "Race to a...
I am spending considerable time researching, studying and gathering CF news items and sharing important findings on our Facebook and Twitter pages. I want to start bringing in the main items of interest into a weekly blog with outbound links to the different news sources. Please like and share...
A must read for all: http://www.scientificamerican.com/article/cystic-fibrosis-might-be-2-diseases/
What could be interesting moving forward in the drug development field for CF is that Lung CF is only half (or much less!) of the story for Pharma incentive into this "rare disease" market. The...
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