Topics

Question #1: Is it PTC 124? / Question #2: Amendment to my question
[Note from ECORN-CF: This combines two questions the questioner asked consecutively.] Dear expert team, I have three important questions: 1) My doctor, who unfortunately is not there anymore now, had me examined in order to find out which mutation I have. I do not remember the name now; ...
28.02.2012
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 ...
27.02.2012
VX-770
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 ...
23.01.2012
What is afoot?
vx-770, vx 809-miglustat, ptc124, DNAase, all these are related to CF, but how are things coming along? What are all these? How has the financial crisis these last two years affected the research? Is gene therapy still being researched, or has it been abandoned? Would it be possible to make custom ...
23.01.2012
Gene therapy for CF
I would like to ask the experts about the course of the research in relation to the gene therapy of CF that takes place in the UK and USA and if there is ground to hope for a medical breakthrough.
23.01.2012
VX770
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 ...
16.01.2012
new inhalative treatment
Hello my daughter's doctor told her that a new aerosol would be placed on the market in early 2012. Can you tell me a little more? Is it an antibiotic, a mucolytic, ..... Thank you in advance for your reply.
02.01.2012
advanced research
hello, I wanted to find out the research against cystic fibrosis, which projects could emerge soon? and then at what stage is gene therapy? thank you
02.01.2012
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 ...
05.12.2011
Ataluren® project (PTC124)
Hello Could you please give me some news about Ataluren® project and if results will be available soon ?
24.11.2011
<<  < 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9 | 10 | 11 | 12 | 13 | 14 | 15 >  >>