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!
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
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 ...
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 ...
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
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 ...
Cystic Fibrosis
What are the new therapeutic perspectives?
clinical trial
Hello Is there any clinical trial regarding patients with mutation G542X/Delta F508 ? Thank you
Ivacaftor and Lumacaftor combination
Hello, Could you tell me if this combination (Ivacaftor/Lumacaftor) has a marketing authorization and whether it is already prescribed by the CF Centers for delF508 mutations? Thank you,
Research for F508del heterozygous
Hello, I would like to know about the research for people with CF with only a single gene of this type (F508del). Thank you in advance
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