Topics

Orkambi®, temporary authorization for Use
Hello, A cohort Temporary Authorisation for Use was granted for ORKAMBI® (beginning of ATU: December 2015, ...
27.02.2016
Lynovex (Cysteamine)
Dear team, while surfing in the Internet, I came across the above mentioned candidate that is acutally in Phase IIb. I was not able to find anything about it on the German CF websites. The pharmaceutical newspaper [German newspaper] reported in January 2015: “The experimental antibacterial ...
14.01.2016
Ataluren® and Bramitob®
I have CF and the mutations F508del & L732X. I am 29 years old and I take for many years Bramitob® (Tobramycin for inhalation). Can I take Ataluren®?
14.12.2015
Ataluren
Is the new medication Ataluren appropriate for administration for the mutation G126D?
11.12.2015
Which mutation has more need for therapy?
Dear expert team, my son (4 years, CF) has the following known mutations: delF508 (inherited by me) and on the other allele R1070Q-S466X. The R1070Q is a missense mutation, the S466X is a nonsense mutation. In case of this rare combination, a marked pancreatic insufficiency has to be expected, ...
09.11.2015
Gene editing
Hello, The last magazine edited by the French CF patient organization indicates that genetic editing techniques, for correcting the genetic material in a relatively simple and inexpensive way, have been developed. They allow to avoid vectors and correct cells directly. Could we have more ...
12.10.2015
Hope for cure
I am a carrier of two mutations, G542X and 2183AA>G. Are there any grounds for hope for a cure in my case? If yes, what does it mean and what will change in my everyday life? If not, is there anything being done for my mutations, or are they rare and indifferent to the researchers?
19.09.2015
Treatment for CF
Is there a treatment/cure for a person with the mutations DF508 & 621+1G>T?
14.08.2015
Heterozygous F508del/2789+5G>A
Hello, I am 56 years old and CF compound heterozygous F508del / 2789 + 5G> A. I read several foreign sites that ivacaftor was prescribed to patients with the same mutations than me, with results that seem spectacular. My pancreas hardly works, but being still pre-diabetic I try to save the few ...
05.08.2015
F508del Heterozygous
Hello, I read this article very encouraging: http://www.news-medical.net/news/20150519/Two-drug-combination-improves-lung-function-in-some-cystic-fibrosis-patients.aspx But for heterozygous F508del, do not we say that if we correct a mutation, we "cure" the disease ?? So, those who have at ...
05.08.2015
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