Topics

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
Duchenne muscular dystrophy and PTC124
M sun age 10 has Duchenne muscular dystrophy. Please how can I get the ptc 124 drug?
23.07.2012
results regarding Ataluren project
Hello, I am the father of a one year old CF girl with a stop codon mutation and I received by mail the first results of phase 3 Ataluren (PTC124). All information that I received are in english, can you enlighten me on these results?
23.07.2012
394delTT and Ataluren (PTC124)
My two children (aged 6 months and 2.5 years old) have cystic fibrosis and one of their mutations is the class I frameshift mutation 394delTT (a.k.a., the scandinavian mutation). 394delTT encodes a stop codon in exon 4.
09.07.2012
Kalydeco (VX770/Ivacaftor)
Have just read the devastating news on the CF Trust Forum that NICE (National Institute for Health and Clinical Excellence in the UK) are not going to recommend the National health service (NHS) provide Kalydeco in UK and want to know your thoughts and any suggestions on this to get it available? ...
09.07.2012
VX770 and R347P
Dear Team, I refer to the question you have already (partly) answered before. I think the more concrete questions is: if VX770 will help R117H - how likely is it that it can work on R347P (or any other class 4 mutation which is not a gating/class 3 mutation)? It is difficult to understand the ...
09.07.2012
genetic therapy
Hello, I wanted to have news about research on gene therapy
25.06.2012
G542X
Recently we were informed that the child I am about to give birth to will carry the mutations PG542X & C2183AA>G. Are these mutations grave? In average what is prognosis? Will the new drugs help my child? Is there some other way to help my child, other that physiotherapy and visiting the CF center? ...
02.07.2012
Meveol®
Hello I have a 4 month old baby suffering from cystic fibrosis. I have heard about Meveol® as a treatment that would be quite effective against recurrent infections. Could you give me some news regarding the research about this drug. Will this drug be available for children? Thanks in ...
11.06.2012
Mutation-specific treatment options
Dear expert team, my son is heterozygous IVS8-2A>C (intron 8) and F508del (exon 10). According to the CF clinic, he has had a mild course of the disease and is in very good shape. Which therapy forms (mutation-specific) are options for this very rare heterozygosity? I assume research mainly ...
05.06.2012
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