Topics

Stem Cells
Hello, Our first son is affected by Cystic Fibrosis. Currently I am pregnant again and heard about archival storage of stem cells from the umbilical cord. Is there really any sense in investing a lot of money there? Of course we would hope that science might be thus far advanced one day that our ...
13.11.2008
PTC-124
Could you please give me some information about PTC-124. My children have the mutation F508/R553X. PTC-124 is according to my research only applicable for stopp-mutations. As a consequence also to the ones of my children? How far is the research on this field really?
16.06.2008
Genotype F508/R553x, Gentamycin Inhalation
Hello, My 30-year old son has the genetic constellation delF508 and R553X. He inhales since approx. 20 years Colistin, after his Pseudomonas showed resistance against Gentamycin. Could he, nevertheless, inhale with Gentamycin? According to Professor Kerem, the substance PTC 124 is also ...
21.01.2008
uridintriphosphate
Hello, In the book „Cystic Fibrosis in Practice“ by „Věra Vávrová et al.,“ there is a note (p. 64) that a good combination for inhalation is amiloride with uridintriphosphate. Among other things it is mentioned that uridintriphosphate is not available here. I would like to ask whether ...
21.07.2008
Genetic therapy in Cystic Fibrosis
Dear expert-team, many people are talking about exchange of genes / genetic therapy. I would be interested in the latest research; what can be done by this method so far? Many greetings,
05.11.2007
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