Topics

research
Hello I want to know if there are researches for people carrying one F508del mutation associated with other mutations, because we heard about Vertex’s trials but they apply only people with two F508del mutations. Thank you for your website.
08.07.2013
F508/R1162X
Dear ladies und gentlemen, unfortunately we have to assume that our unborn son (31 st week of gestation) has inherited the mutations F508 and R1162X, as we are both each heterozygous carrier of one of those mutations and in the last prenatal ultrasound investigations always an echogenic bowel ...
19.06.2013
CF 2013
Essence of question: is there any information of new medicines improving the underlying defect in CF?
27.05.2013
Inhalation of sodium bicarbonate
Dear expert team, I would like to know in which cases it is recommended in CF to inhale NaHCO3 (sodium bicarbonate), how this is done (amount/frequency/dosage/kg or age) and if this inhalation is done in addition to the inhalation with NaCl-solution or instead of it (i.e. in addition with ...
15.05.2013
Bronchitol® benefit
Bronchitol® is proposed to CF patients. Is the treatment added to inhaled hypertonic saline serum/ inhalation with salbutamole or does it subsitue the treatment? What about differences between lipromatase and CREON®?
13.05.2013
Kalydeco® also before 6th birthday?
My son, 4, CF, has the mutations G551D and dF508. Until now he is doing quite well concerning his general condition, even if he already had to fight strongly in his first year of life. Now I am asking myself if Kalydeco® can be prescribed for him even before his 6th birthday. Finally it is ...
10.05.2013
VX983
Hello After VX770, VX809 and VX661, VERTEX announces a clinical trial with VX983 (in combination with Kalydeco™-VX770). This clinical trial concerns patients homozygous for deltaF508. Could you tell me more? Could you tell me if VERTEX has made the deltaF508 mutation its "workhorse" or if ...
10.05.2013
Splicing mutations
Do you know whether there are or there will be clinical trials on CF patients carrying a splicing mutation (other than stop-codons)? Additionally, can you tell me whether VX770 (for G551D mutations) and VX770/VX809 (for homozygous F508del mutations) will be further tested in patients with any kind ...
07.05.2013
MEVEOL® studies
Hello Few times ago I discovered the MEVEOL® program, performed by the pharmaceutical company ALAXIA. One publication has been done in 2009. On the ALAXIA’s web site we can noticed that: “ - MEVEOL® is a “candidate drug” effective for treating respiratory infection in cystic ...
03.05.2013
Future of gene therapy in CF
Hello. We don’t hear much talk any more about gene therapy and this treatment seems outdated ... Can you tell me a little more: what are the tracks to come in this area? Are there special difficulties to operate this way? Is this therapy still a hope for patients with cystic fibrosis? Thank ...
03.05.2013
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