Kalydeco (VX770)
Have just read the devastating news on the CF Trust Forum that NICE are not going to recommend the NHS provide Kalydeco in UK and want to know your thoughts and any suggestions on this to get it available? Obviously as much media coverage as possible I imagine. Whilst NICE relates to England and ...
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 ...
Aberrant Splicing Mutations and Ataluren
My 14 year old daughter has the two mutations DF508 and 621 + 1G > T. It is my understanding that the 621 + 1G > T mutation causes aberrant splicing of mRNA. How is that different from a nonsense mutation? Currently, I see that Ataluren trials are targeting nonsense mutations in Class I.  ...
vertex 770 and r347p
Dear team, my daugther FC has got mutation r347p (class iv). I red that vertex 770 could work on r117h (class iv too). Is the defect of conductance caused by r347p similat to the defect caused by r117h? Is it probable that vertex 770 can work also for r347p? In case it works for r117h, will it ...
Hi there, My daughter is currently 7 months old with G551D as one of her mutations. I just wondered if the drug is approved this year in the 6yrs and older subset can it then be used in younger children with the dosage ammended accordingly? Or would they need to carry out a clinical trial in ...
VX770 - Kalydeco
The child in question has G551 and F508 Delta mutations. For us VX770 provides great hope and just pray soon such a drug will be available to all other CF patients. I have read as much as possible on this drug but do not see information that I would wish to know. As a person with one faulty ...
I know although VX770 is probably soon to be licensed it is still very early days. However, what I have read is that this should considerable help a CF patient with G551 gene. PRESUMING this medication opens the 'gate' it is said that although not a cure this should control CF in the same way that ...
Vertex-770 potentiator
Dear expert team, my daughters genotype is DF508 and R347P, now that the drug for G551D is going forth for approval because of its effects on the gene do you have any further data to support that this drug would also be effective on other class IV types like R347P as I know it has some effect on ...
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 ...
PCT 124 Trials
Could you possibly let me know about the progress on the PCT 124 drug, if any? Regards Tandey Muller"