Topics

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
GLPG1837 Galapagos clinical trial
Hello A clinical trial with the CFTR protein potentiator GLPG1837 (Galapagos) has just started. Obviously this molecule is for patients carrying a class III mutation. Is the clinical trial ongoing in France? When will results be available? If compared to Kalydeco® could this new molecule ...
20.06.2016
Glutathione
Hello Could you please give us information regarding reduced Glutathione? Is there any deficiency for CF patients (that would be expected) ? Is this deficiency associated with some symptoms in particular? Is there any research done with external supply of Glutathione? What type of Glutathione? ...
06.06.2016
Orkambi®
Good evening, I have read the side effects of Orkambi®. What are the results of studies in countries that prescribe this treatment for a couple of years now (regarding these side effects)? Best wishes
13.05.2016
MUTATION c.1680-886A>G (remainder)
Hello, Thank you for your reply on the mutation c.1680-886A> G. It is therefore a splicing mutation and not a "nonsense" mutation. However, since due to reading frameshift it leads to a stop codon, can we assume that patients with this mutation would be eligibe to ataluren (PTC124)? Thank ...
11.05.2016
Contraception in case of the intake of Orkambi® or the like
My daughter (20 yrs) takes part in a Vertex study, the drug is similar to Orkambi®. For contraception, the pill and all other hormone-based devices, are not suitable. How can she do an effective contraception without being afraid of getting pregnant? The gynecologist has no understanding for her ...
25.04.2016
Orkambi® Side Effects
Good evening Do you have information on the side effects of this treatment (Orkambi®) available in France since shortly but already widely distributed in CANADA or the USA? The side effects are severe and there is little feedback to take a decision. Best regards
24.04.2016
Sweat test with Kalydeco®
Dear physicians, my son has the mutations G1244E and R553X and gets Kalydeco® for 2 months. He has had a sweat test result of 110 at his diagnose about 2 years ago and now after two months of Kalydeco® the value went down to 80. My question is, do you think that this is the endpoint or can one ...
16.04.2016
Healing of genetic disorders?
The university of Dresden (Germany) together with the Heinrich-Pette institute in Hamburg, Germany, developed a method, that can liberate the HIV code from the human genetic material. It is reported, that with that there is a chance, to heal genetic diseases, as with this method one could influence ...
16.04.2016
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