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The first baby has been born in Ireland where a technique called Pre-Implantation Genetic Diagnosis (PGD), was applied to screen embryos from the mother for suspected genetic conditions such as cystic fibrosis. After screening, the identified healthy embryo is then implanted back into the...

A 76-YEAR-old woman diagnosed with cystic fibrosis, has become the oldest ever first presentation case in Ireland. US and UK registry data reveal the oldest patients with CF in these countries are 82 and 79 years respectively. An increasing number of Adults are being diagnosed later in life due...

ProQR Therapeutics, a Dutch biotech developing mRNA treatments for CF, announced its IPO terms today. They have developed a technique that targets and repairs defective messenger RNA (mRNA) and treats the underlying genetic mutation for CF, which they believe is superior to small molecules...

Vertex will be presenting some important data at the upcoming NACFC conference in early October. Combination trials (Kalydeco + lumacaftor) in people with CF who are homozygous for F508del-CFTR. Presentations are expected to include previously announced data as well as new interim data from...

Some Cystic Fibrosis related highlights in the news this week: FDA Grants Fast Track Designation to Aradigm’s Pulmaquin for Non-Cystic Fibrosis Bronchiectasis http://t.co/HZHT0cgu6s Scientists aim to develop rapid test system for immune dysfunction to treat cystic fibrosis...

Please help Tim Wotton win the People's Book Prize. His incredible CF memoir ‘How have I cheated death?’ - A story of triumph over adversity, this compelling chronicle, the first book written by a CF sufferer at 40, offers hope and inspiration, demonstrating what cannot be cured needs to be...

Promising results from a gene therapy study late last year by Hans Clevers, a Dutch stem cell researcher, showed that a technique called CRISPR can be used to correct the CFTR gene defect associated with cystic fibrosis in human stem cells. Using cultured intestinal stem cells, they were...

A BREAKTHROUGH has allowed scientist to discover that adults with Cystic Fibrosis may lack a very important cell surface molecule that is crucial for proper immune system defense. The scientists found that the immunological cell surface molecule HLA-DQ is reduced or absent in many people...

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...

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...

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...

Just came across this: Read full article here.