Topics

Lungs or pancreas - clinical trials
Is there anything for the mutations ending in ...X. When will it be available in Greece? Does it concern the lungs or the pancreas. Thank you!
18.02.2011
Q493X
My daughter has just had Q493 identified as her second mutation. (She also has D508) Can you tell me anything about Q493 as it seems fairly rare?
11.01.2011
Hyaluronic acid
Hello, on the 1st of September it has been in the media, that hyaluronic acid plays an important role in CF. (http://www.idw-online.de/pages/de/news384304). Can this give rise to hope or is it rather uninteresting for the patients? Many thanks for your answer.
05.10.2010
PTC124 study
Hello, I heard about a PTC124 study. My daughter is five years old and has the R553x/IVS8-5T-TG12 mutations. Could the above medication help her? How far advanced is the study by now? Many thanks. Kind regards C.M.
07.09.2010
PTC 124
Is it true that PTC 124 is effective with class 1 mutations? That is, with the cftr dele2,3 mutation? Thanks
06.09.2010
Current research of gene therapy
Hello, I would like to know how the current state of research of gene therapy in the field of cystic fibrosis is? What are the prognoses for the future, especially for newly diagnosed babies? And if other countries are “more advanced” in comparison to Germany? Many thanks for your ...
23.06.2010
Vertex-770 potentiator
Dear expert team, my son's genotype is DF508 and R347P. As VX-770 only studies effectiveness in G551D (and DF508) at the moment I wonder what the probability is that this drug also works for other (class III-V) mutations with a certain functionality of the CFTR protein (my son must have some ...
05.01.2010
Drug approval USA Germany
Dear expert team, There are promising new medicaments for the treatment of Cystic Fibrosis in the drug development pipeline, among these, substances of Vertex. Just in case these drugs will be approved, how likely is it in your opinion that these Vertex substances (and others like Denufosol and ...
05.11.2009
Corrector- and Potentiator-drugs and male fertility
Dear expert team, do correctors and potentiators such as VX 809 and VX 770 influence presumably the male fertility in case of delta F508 being positive? I do not expect a binding answer - assumably there will be no tests available at the moment - only if these new drugs could have theoretically ...
02.11.2009
Mutation F508del/E822X
My daughter is six years old and has CF – mutation F508del/E822X. Is there any progress in the treatment of this specific mutation and does it matter if the manifestation of the symptoms of the said mutation is lighter?
29.12.2009
<<  11 | 12 | 13 | 14 | 15 | 16 | 17 | 18 | 19 | 20 | 21 | 22 | 23 | 24 >  >>