Topics

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
PTC 124 / Vertex VX-809
My son has the following mutations: 1717 1g --> A (class I mutation) as well as s549R (class II mutation). “1717 1g --> a” is a class I mutation. More precisely, it is a splice site mutation which, however, triggers a stop codon. Is PTC 124 effective with this kind of mutation? The ...
24.03.2009
CF with two nonsense mutations
Hello, our son (5 weeks old) was born with meconium ileus and suffers from exocrine pancreatic insufficiency, which is being treated with pancreatin. By now, it is clear that he has CF. The genetic test showed a compound heterozygosity with nonsense mutation in exons 6 and 11 (heterozygous ...
02.03.2009
PCT 124
Dear expert advice team, I have the following mutations, 508 and R 1066C. Since R1066C is a so-called nonsense mutation: Could you inform me if PCT 124 can be a cure in this case or makes treatment more effective? Is there already a date when this drug will be available? Thank you very ...
24.02.2009
PCT 124
Does PCT 124 help in case of cftr dele 2,3?
24.02.2009
Information on PTC-124
Hello, I have read with great interest the question of a father of two CF children in Austria. In this question, the study drug PTC124 was mentioned. Could you please provide more information on this interesting medication and scientific approach? My daughter (28yr) suffers from CF, which was ...
15.01.2009
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