Topics

Current research of gene therapy
Hello, I would like to know how the current state of research of gene therapy in the field of cystic fibrosis is? What are the prognoses for the future, especially for newly diagnosed babies? And if other countries are “more advanced” in comparison to Germany? Many thanks for your ...
23.06.2010
Vertex-770 potentiator
Dear expert team, my son's genotype is DF508 and R347P. As VX-770 only studies effectiveness in G551D (and DF508) at the moment I wonder what the probability is that this drug also works for other (class III-V) mutations with a certain functionality of the CFTR protein (my son must have some ...
05.01.2010
Drug approval USA Germany
Dear expert team, There are promising new medicaments for the treatment of Cystic Fibrosis in the drug development pipeline, among these, substances of Vertex. Just in case these drugs will be approved, how likely is it in your opinion that these Vertex substances (and others like Denufosol and ...
05.11.2009
Corrector- and Potentiator-drugs and male fertility
Dear expert team, do correctors and potentiators such as VX 809 and VX 770 influence presumably the male fertility in case of delta F508 being positive? I do not expect a binding answer - assumably there will be no tests available at the moment - only if these new drugs could have theoretically ...
02.11.2009
Mutation F508del/E822X
My daughter is six years old and has CF – mutation F508del/E822X. Is there any progress in the treatment of this specific mutation and does it matter if the manifestation of the symptoms of the said mutation is lighter?
29.12.2009
CF research
Are there any tests done in Belgium to cure people with CF ?
13.07.2009
PTC 124 Trials
Could you possibly let me know about the progress on the PTC 124 drug, if any? Regards Tandey Muller"
09.07.2009
PTC 124
MY 8 YEARS OLD DAUGHTER HAS THE FOLLOWING MUTATIONS, DELTA F 508 AND W1282X. IS PTC 124 EFFECTIVE FOR THIS KIND OF MUTATION? AND WHICH ARE THE RESULTS OF THE LAST CLINICAL TRIALS? DO I HAVE SOMETHING TO HOPE FOR?
18.06.2009
pct drug
hello, i would like to know if the pct drug can be used on haemophiliacs.
29.04.2009
VX 809 -gene therapy
My son is 6 years old and he has a cystic fibrosis - genotype Delta F 508 in both allels. When we can to expect the gene therapy of product VX 809 in Europe as part of necessary medical therapy?
07.04.2009
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