Topics

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
Lungs or pancreas - clinical trials
Is there anything for the mutations ending in ...X. When will it be available in Greece? Does it concern the lungs or the pancreas. Thank you!
18.02.2011
Q493X
My daughter has just had Q493 identified as her second mutation. (She also has D508) Can you tell me anything about Q493 as it seems fairly rare?
11.01.2011
Hyaluronic acid
Hello, on the 1st of September it has been in the media, that hyaluronic acid plays an important role in CF. (http://www.idw-online.de/pages/de/news384304). Can this give rise to hope or is it rather uninteresting for the patients? Many thanks for your answer.
05.10.2010
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