Topics

Colchicin
Hello, I read an article (relatively old) that speaks of colchicine, an anti-cancer molecule which could have beneficial effects on CF. Could you please give me some news about this molecule?
05.05.2011
Bronchitol
The new drug bronchitol is already available in Australia. Will it be available on the European market soon?
28.04.2011
NB-401
I vaguely heard of NB-401. It is an aerosol that would combat bacterial strains highly resistant to current treatments. Could you tell me a little more? ongoing studies? Thanks a lot for your response
12.04.2011
VX-661, for patient with the F508del mutation
Regarding the phase II study of VX-661, for patient with the F508del mutation (deltaF508), which should begin during the second semester 2011: - Is it the same study than the one where VX-770 is used in combination with VX-809? Or is it a different study? - Is study is also specific for patients ...
12.04.2011
Miglustat and VX-809
Hello, What are the news regarding Miglustat ...Are you aware of new trials ? On the other hand, when will the results of the study on VX-809 be availbale ?
12.04.2011
Research on Meveol®
Hello, I attended this weekend in Reims the annual General Assembly of Vaincre la Mucoviscidose. In the presentation on advances in research, it has not been referred to the ongoing development of the drug Meveol® whose lab results were announced effective and promising against bacteria ...
07.04.2011
Compound heterozygosity - does one or both defects have to be corrected causally?
Hello, my daughter, five weeks old, born with meconium ileus, genetic result: delta F 508 on one allel and a stop-mutation on the other allel, has therefore a heterozygote combination of 2 different defects. According to my research the substance VX809 is investigated for delta F 508 and the ...
07.04.2011
Mutation
Hello, Is the 1717-1G> A mutation (which is part of class I mutations) associated with premature discontinuation of protein production secondary to a stop codon? In this case, could it be accessible to PTC124 (Ataluren)? Many thanks for your reply. Yours.
29.03.2011
Composite CFTR heterozygote
Hello, We are the parents of an 11 years old CF boy. He has 2 different CFTR mutations from class II and I respectively: F508del and 1717-1G>A. If a treatment like the Vx809 is efficient for the F508del mutation, could our child be “cured”? Would it be necessary to add another treatment for ...
28.03.2011
Molecules and mutations
Hello Main research studies focus on personalized treatments. But could you specify if - In case of success, will Miglustat and Vx809 be effective only on f508 mutation or all class 2 mutations as d507? - Could PTC124 be effective on the New1 class mutation 1811+1,6 Kb A>G (splicing mutation ...
24.03.2011
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