Topics

RNA Therapy
Hello, There is gene therapy, protein therapy and now RNA Therapy. What is it about ? And above all, would positive results in RNA therapy address all patients (such as gene therapy) or would it depend on mutations (such as protein therapy)? Thank you in advance for your answer.
20.01.2017
Ataluren and research
Hello, Some time ago I asked a question concerning Ataluren on this platform. Our two daughters (28 and 31) have the genotype Delta F508 and R1162X. Our CF physician mentioned at the last control, that recently quite promising results have been reported for Ataluren at a CF congress in London. ...
05.12.2016
New curcuma preparation
Hello, As far as I know, the effect of curcuma on CF is tested in the Netherlands in clinical studies. Now I have read, that a new preparation is going to be put on the market, that should guarantee a high bio-availability of curcuma and therefore should be especially suitable for CF ...
12.10.2016
Lactoferrin
Hello, are there treatments or research regarding the use of lactoferrin as a bactericidal substance in cystic fibrosis?
19.09.2016
Research Update
Hello, Could we have a French synopsis on positive results regarding research (Vertex, QR-010, PTC124, others?) for patient heterozygous for the F508del mutation? Thank you.
19.09.2016
Quinsair®
Hello, could I get some information about Quinsair® (inhalation of levofloxacin)? Since when it is on the market, how, where and when is can/should be used? Best regards,
25.07.2016
QR-10
Hello Which mutations are concerned by the QR-10 molecule which targets the correct synthesis of CFTR protein? Thank you for your reply
25.07.2016
CTP-656 POTENTIATOR
Hello Could you tell us which company produces the activator CTP-656 and which patients will potentially benefit from this molecule? Thank you for your reply
25.07.2016
Meveol®
Dear expert team, I read something about the development of Meveol®. It seems to be very promising to me. Is there anything new at the moment about this topic? Can one make a prognosis, when the drug will be on the market? Many thanks in advance.
16.07.2016
Mutations F508del and R553X
Dear expert team, Our son has been diagnosed to suffer from CF at the beginning of January at the age of 9 weeks, after we had been admitted to hospital with a hemoglobin value of 6. The little boy became 2 blood transfusions and takes zinc tablets since then, as he also had a zinc deficiency. ...
24.06.2016
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