Topics

SPLUNC1 protein
Hello Could you give some information concerning SPLUNC1 and its effects in cystic fibrosis? Do we know well this protein or is it only the beginning of the research? Are there studies planned ? Thanks for your answer.
14.11.2012
Nonsense mutations
Hello, I am a mother of a child who has a nonsense mutation and I would like to know whether, apart from Ataluren, there were other current researchs on these mutations. Thank you in advance.
13.11.2012
Mutations
Could you give me some information about these 2 mutations, G551D associated with Q1476X : which class do they belong to, which respiratory or digestive problems can they cause, severity of the disease, even knowing that the disease is not similar between subjects. Thank you in advance for your ...
22.11.2012
Combination therapy: Kalydeco+VX809
Following recent results presented at the NACFC in Orlando, it seems that the association Kalydeco+VX809 is promising. Will the drug be commercially available in 2015/2016?
12.11.2012
Genetics IVS8T5 - Delta F 508
Which treatment options exist for this specific genetic situation? And can one reckon with the development of specific drugs? Kind regards.
29.10.2012
ataluren
Hello Do you have information about the next steps in the ataluren development? When will the open study be completed ? And when will we have the results? Thank you for your site
25.10.2012
Ataluren with stop mutation R1162X
Unfortunately the latest results that were published at the CF congress in Dublin are not really convincing. I have the following question nevertheless: Is it true that the R1162X stop mutation is suited very well or at least better than other stop mutations for therapy with Ataluren? Were ...
11.09.2012
Why are Ataluren results considered disappointing?
[In this Q/A two questions were summerized as the given answer was the same, both questions can be read here] Hello, I read about the phase-III study on Ataluren as well as the PTC press release on the website of the German CF patient association, and I do not really understand why the ...
11.09.2012
Vertex® agent for 18 patients realistic?
Dear expert team, For some time now, I have been following Vertex® (via their website), who have successfully completed clinical trials for a mutation-based therapy for ca. 4% of CF patients with KalydecoTM. KalydecoTM was now approved in Europe as well on July 27. I am happy for the 1500 ...
13.08.2012
CF with F508del and G542X mutations
Hello, My son was born in april 2011. He was diagnosed with CF (two mutations G542X and F508del). Are there any progress on treatments that could cure these two mutations? He never had any diseases, it receives his Physio and takes Creon, the ADEK vitamins and does not gain much weight. Yours
13.08.2012
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