Topics

DeltaF508/R553X, part II
Dear expert team, Please allow me a further question concerning the genotype mentioned in the subject line. In “Ataluren (PTC124) induces cystic fibrosis transmembrane conductance regulator protein expression and activity in children with nonsense mutation cystic fibrosis” ...
17.04.2012
VX 770
Hello, A CF individual from France told me that the French health authorities do not give approval for VX 770 (not even via the "named patient program" of Vertex) since it is supposed to cause hepatitis side effects. Do you know more about it? [The second part of the question was only ...
22.03.2012
PTC124 approval?
Dear expert team, as far as I know, the drug is currently in phase III, which is supposed to end by mid-2012. Can you predict roughly how long it will take until the drug is approved and how likely it is to get approved at all? In case it does not get approved, would it be possible to ...
22.03.2012
Ivacaftor US
Hello, the drug Ivacaftor is now being marketed in the United States, is it prescribed routinely to all carriers of the mutation in question? Is it going to be the same in France when it has his license? When can we have details of the concrete effects (hopefully very beneficial) in patients and ...
22.03.2012
VX-770
Hello, The VX770 molecule (Ivacaftor) acts on the G551D mutation and, also, positively effects on 9 other class 3 mutations. Which ones? Thank you for your response.
28.02.2012
vertex 770 and r347p
Dear team, my daugther CF 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 ...
28.02.2012
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
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