Topics

Kalydeco®
Dear expert team, I am a 24-year old girl suffering from CF. I come from Bulgaria, where there is no European standard therapy and no CF-experts either. That means that I get information about new drugs from German and English articles. I have read several times about Kalydeco® (for G551D). I ...
05.02.2013
EMA (European Medicines Agency)
Hello, I would like to know what is the role of EMA in treatment research? Thank you in advance.
21.01.2013
new medicines
What can you tell us about new medicines to be expected? How are the examinations and tests progressing? What are the expectations of the medicines and when are they expected to come on the market? Are they meant for delF508?
10.01.2013
Genetics, diagnosis and therapy option
Can the new drugs (Vertex) only used with F508del homozygous or would that also be possible with F508del and IVS8T5? Can one tell exactly from the genes or through a test (e.g. blood) what our son's "protein problem" is? Many thanks.
20.11.2012
CYSTIC FIBROSIS
What is new in the field of gene therapy research except of routine treatment with antibiotics, enzymes and lung transplantation?
20.11.2012
VX Combo trial 770/809
Hello, how long will the combo trial phase III last? When will the results be presented? Thank you, Best regards,
15.11.2012
G551D/G542X
My husband is suffering from CF and carries the mutations G551D/G542X. Do theses mutations express themselves within the cells or does one mutation dominate the other ?
14.11.2012
SPLUNC1 protein
Hello Could you give some information concerning SPLUNC1 and its effects in cystic fibrosis? Do we know well this protein or is it only the beginning of the research? Are there studies planned ? Thanks for your answer.
14.11.2012
Nonsense mutations
Hello, I am a mother of a child who has a nonsense mutation and I would like to know whether, apart from Ataluren, there were other current researchs on these mutations. Thank you in advance.
13.11.2012
Mutations
Could you give me some information about these 2 mutations, G551D associated with Q1476X : which class do they belong to, which respiratory or digestive problems can they cause, severity of the disease, even knowing that the disease is not similar between subjects. Thank you in advance for your ...
22.11.2012
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