Topics

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
Function of correctors for stop mutations
Dear ladies and gentlemen, I would like to know, how exactly the correctors (e.g. Lumacaftor) are acting on stop mutations on the molecular genetic level. Are those synthetic tRNAs that bind to the wrong stop codons? I would be pleased if you could explain the function of the corrector ...
16.04.2016
Replace a gene with a special method
Hello, is the CRISPR / cas9 method envisaged in cystic fibrosis? Are trials going on? Thank you
08.04.2016
Mutations F508del & I507del
Hello, We are the proud parents of a beautiful boy with the mutations F508 and I507. We would like to have more information on both mutations together. Our son’s disease looks more digestive than respiratory, although he already had two pseudomonas colonizations (halted by an oral cure and an ...
27.02.2016
CF Treatments
Hi, I'm a CF, I am 15 years old. I am looking for new drugs. I'd like your response. Thank you.
27.02.2016
Orkambi® prescription
Hello My son aged 30, has a F508del mutation but has not been offered ORKAMBI® yet. His FEV1 is 27% and he made all pre-transplant tests a year ago. Are all patients taken into consideration for Orkambi or are there cases where this treatment is not recommended? Has he to request it from the ...
27.02.2016
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
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