Topics

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
H199Y mutation
Hello, my daughter born in 1995 has a H199Y mutation not listed in one of the four mutation classes (confirmed by doctor and scientist of the French CF association). How is this possible and how to know in this case if one of the latest treatments and future developments will be of ...
05.08.2015
Gene therapy: GL67A
Hello, The results published in The Lancet Respiratory Medicine by British researchers are positive although modest ... and this news delights us! Could you tell me if, contrary to the protein therapy, gene therapy would be beneficial for all patients or the type of mutation also will affect the ...
01.08.2015
Kalydeco® and salt supplementation
Hello, In case of scorching heat salt supplementation is recommended for all CF patients. But what about for patients on Kalydeco®? Because their sweat normalizes, so we can assume that salt losses are less ...
01.08.2015
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