Topics

Orkambi®
Hello Has an Orkambi® marketing study been scheduled for heterozygous F508del CF patients? (Excluding gating mutations).And in children under 12? Can you send me the link of the site or sites that allowed you to answer me please? How have other mutations been tested in phase 2? Per class? case ...
01.08.2015
Off-label use of Kalydeco
Dear physicians, I am a mother with a 3.5-year-old son suffering from CF, living in Germany. My son has been diagnosed at the age of 2.5 years. Overall, his health condition is good. However, the illness affects markedly the gastro-intestinal tract. He has had a long-lasting colonization with ...
28.06.2015
Kalydeco off label
Hello, my daughter has the mutations delF508 and D1152H. How great is the chance to get Kalydeco off-label? There are obviously in-vitro studies that show, that the CFTR channel is functioning nearly completely in case of intake? There is already a study from Denver about this? In the USA human ...
18.06.2015
Genetic therapy heterozygous DF508
Hello, how far is research concerning DF508-heterozygous patients? Are there first study results, when can one expect a licensing in case of promising results? Thanks!
12.06.2015
Delta F508 effective 2015
Hello, my 12-year-old daughter suffers from the above mentioned mutation. I have heard, that there is a drug available in the course of this year. Is it true and how does it act? Thank you
04.06.2015
G551D/S549R Kalydeco® - yes or no?
Hello! Our daughter has been diagnosed lately at the age of 6 years. She has a marked pancreatic insufficiency, for 1.5 years a stagnating course of height and weight, furthermore an FEV1 value of 111%. Staphylococcus aureus and Haemophilus influenzae have been there for a short time, however have ...
16.05.2015
Phage therapy
Dear ladies and gentlemen, in the past, it has been written quite a few times about this topic, but as some time passed inbetween, I ask myself, if there are important findings. My husband (30, CF), has for about 10 years the germ Achromobacter xylosoxidans in the sputum. This germ did until ...
16.05.2015
Acting mechanisms of Ivacaftor and Lumacaftor
Hello, I have a question about the acting mechanisms of Ivacaftor and Lumacaftor. In how far do they change the molecule respectively which biochemical processes are involved? Best regards
06.05.2015
Genotype F508del/1717-1G>A and clinical trial
Hello, First thank you for your site and your answers! Here's what I read in the search results. "Currently, four Phase 3 studies are underway for VX-661 (100 mg once daily) in combination with ivacaftor (150 mg every 12 hours). One of those studies is in people with two copies of the F508del ...
06.04.2015
Cystic Fibrosis
What are the new therapeutic perspectives?
13.03.2015
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