Topics

H199Y mutation
Hello, my daughter born in 1995 has a H199Y mutation not listed in one of the four mutation classes (confirmed by doctor and scientist of the French CF association). How is this possible and how to know in this case if one of the latest treatments and future developments will be of ...
05.08.2015
Gene therapy: GL67A
Hello, The results published in The Lancet Respiratory Medicine by British researchers are positive although modest ... and this news delights us! Could you tell me if, contrary to the protein therapy, gene therapy would be beneficial for all patients or the type of mutation also will affect the ...
01.08.2015
Kalydeco® and salt supplementation
Hello, In case of scorching heat salt supplementation is recommended for all CF patients. But what about for patients on Kalydeco®? Because their sweat normalizes, so we can assume that salt losses are less ...
01.08.2015
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
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