Topics

Kalydeco® for children
Dear expert team, is there any concrete information about the time of licensing of Kalydeco® for children from 2-5 years? How successful were the studies? My daughter has G551D, can the health insurance in spite of this refuse to pay the costs? Are there any reasons that speak against ...
03.01.2015
Clinical trial genetic therapy
Hello Do you know if the UK clinical trial "genetic therapy" is closed and what are the first conclusions? Thank you
10.12.2014
Mycobacteria
Hello, My 6 year old son just had a sputum culture that shows the presence of mycobacteria. I wished to have more information on this germ and whether my son will have this germ all the life as I gather that it is resistant to antibiotics. Also I'd like to know where is the research regarding ...
03.12.2014
Correctors and germs
Dear expert team, supposed one of the new drug combinations e.g. by Vertex would be suited for a correction of the homozygous Fdel508 defect, would encourage the cells to take up their tasks to at least 10% until e.g. 40%, and would result in a similar improvement concerning FEV1 and ...
25.11.2014
G542X mutation
I am 19 years old, have CF and carry the mutation G542X. I read and hear about a medication that will be commercially available the coming years for the radical cure of the disease. Is this medicine available to purchase? If not, do you know what state it is in?
03.11.2014
R1162X and Q1035X
Hello dear expert team, recently, the mutations R1162X and Q1035X have been detected in my genetic material. Could you please answer the following questions about this combination: - Is there a prognosis concerning the course of the illness? - How is the frequency of both mutations ...
03.11.2014
Compassionate use - VX809
Hello, I turn to you with a question concerning VX-809. Vertex reported at the presentation of data (Phase 3 Ivacaftor / Lumacaftor from June 24th, 2014) about a "compassionate use" program [comment of the translator: a compassionate use program describes the tolerated usage of a drug that is ...
13.10.2014
Compassionate use program for deltaF508 patient
Hello In the press release dated on June 24th, 2014, the Vertex company talked about a "compassionate use program” [comment of the translator: a compassionate use program describes the tolerated usage of a drug that is not licensed for humanitarian reasons] for people who really need a new ...
06.10.2014
Treated with Kalydeco®. And then?
Hello, patients receiving a treatment like Kalydeco®, do they have a different life? I mean by that, do they still need to take many drugs such as Creon®, do they still do a lot of physiotherapy? Or it is case by case? Is there an increase in life expectancy, but is their life a little more ...
15.09.2014
Vertex - results of a phase 2 study for patients heterozygous for F508del
Hello The Vertex company has published results regarding patients heterozygous for the F508del mutation, here is the link: http://investors.vrtx.com/releasedetail.cfm?releaseid=862727. It seems to me that patients heterozygous for the F508del mutation could have a potential treatment like the one ...
15.09.2014
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