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Keywords
- ABPA_Aspergillus
- accompanying diseases
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Topics
- 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
- 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