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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
Ataluren
When will Ataluren be available in Germany?
02.08.2012
Ataluren
Does Ataluren help with the Delta F508 mutation?
02.08.2012
MIGLUSTAT
Hello, The latest results of Miglustat's clinical trials were not those expected. Are researches on this molecule definitively abandoned? Thank you in advance for your response
30.07.2012
VX 770 without effect
Hello, after taking VX770 for four weeks, I (29 years old, FEV 32%) could not detect any measurable or subjective improvement (FEV unchanged and large amount of secretion as usual). Since, apart from having the G551D mutation, I am also G542X carrier, the question is whether this second ...
30.07.2012
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