Topics

F508del heterozygous- first recognitional breakthrough?
Hello, did I get it right from the English that for the first time there has been a significant improvement in the usage combination VX-661 and KALYDECO® (Ivacaftor) in case of F508del heterozygous patients "with/independent" of G551D? That sounds like a breakthrough for the heterozygous ...
28.07.2014
Drugs for delF508
Hello, I have recently visited the annual meeting in Bad Soden, Germany. I have heard there, that Kalydeco® together with a potentiator is said to be licensed already in June 2014. Short-dated also a liquid for children is said to come on the market. Did I get this right? Does this mean the ...
21.07.2014
Results from Vertex clinical trial for patient with delF508 mutation
Hello I’m a CF patient and I’m carrying 2 different mutations: delF508 and 1717-1GA of exon 11. I would like to know if you have the results of the Vertex‘s clinical trials. Are they encouraging? If not, when will they be available? Best regards
14.07.2014
Cayston®
I’ve heard (by a service provider) about a new molecule named “Cayston®” that is used to treat CF adultes againt Pseudomonas aeruginosa. This molecule would be used with a new machine that will arrive on the market (or already has?). This person could not tell me more. I turn to you in order ...
14.07.2014
VX-809 research
Hello, in the research news dated 01.01.2013 (here the link to a German website: http://muko.info/forschung/news-detail-und-archiv/detailansicht/article/zulassungsprogramm-fuer-korrektur-des-f508del-soll-2013-starten/17.html ). it has been said, that the market authorization for "VX-809" ...
09.07.2014
N30 Pharmaceuticals company
Hello Do you have information regarding the progress of research performed on 2 molecules ((n91115 et n6022) from N30 Pharmaceuticals company? Can we have hope that this type of drug could emerge soon? Do you think that these molecules and the miglustat, based on their mechanism of action, ...
26.05.2014
Amlexanox
Dear expert team, I got aware of the following publications: http://www.ncbi.nlm.nih.gov/m/pubmed/22938201/ and http://www.google.com/patents/EP2437741A1?cl=en I would like to know if there is any data yet concerning the correction of CF nonsense mutations? Respectively, if there are any ...
12.05.2014
Nitric oxide therapy
Hello, are there already any news concerning the above mentioned therapeutic trial? Thanks.
29.04.2014
Research on delF508 mutation
Hello It’s not the first time I ask you a question, and first of all I would really thank you for the help, the information and sometimes the comfort that you bring us through this website. I don’t want to abuse your precious time but could you resume in a few lines the research which is ...
28.04.2014
Meveol®
Hello I have just read: « A new treatment against cystic fibrosis could emerge by 2016. This is a project supported by Bpifrance (bank financing and investment) who just released € 10 million for a new medical program called Alipex. Of the three companies involved in research: Alaxia, a ...
15.04.2014
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