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Keywords
- ABPA_Aspergillus
- accompanying diseases
- air-improving devices
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- asthma
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- drugs under development_genetic therapy
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- general aspects
- genetics
- health care
- hepatobiliary disease
- hygiene
- i.v.-lines
- inhalation
- lung
- microbiology
- miscellaneous
- modulator therapy
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- nutrition and GI problems
- oxygen supplementation_therapy
- physiotherapy
- Pseudomonas aeruginosa
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Topics
- Ataluren
- When will Ataluren be available in Germany?
- 02.08.2012
- Ataluren
- Does Ataluren help with the Delta F508 mutation?
- 02.08.2012
- MIGLUSTAT
- Hello, The latest results of Miglustat's clinical trials were not those expected. Are researches on this molecule definitively abandoned? Thank you in advance for your response
- 30.07.2012
- VX 770 without effect
- Hello, after taking VX770 for four weeks, I (29 years old, FEV 32%) could not detect any measurable or subjective improvement (FEV unchanged and large amount of secretion as usual). Since, apart from having the G551D mutation, I am also G542X carrier, the question is whether this second ...
- 30.07.2012
- Duchenne muscular dystrophy and PTC124
- M sun age 10 has Duchenne muscular dystrophy. Please how can I get the ptc 124 drug?
- 23.07.2012
- results regarding Ataluren project
- Hello, I am the father of a one year old CF girl with a stop codon mutation and I received by mail the first results of phase 3 Ataluren (PTC124). All information that I received are in english, can you enlighten me on these results?
- 23.07.2012
- 394delTT and Ataluren (PTC124)
- My two children (aged 6 months and 2.5 years old) have cystic fibrosis and one of their mutations is the class I frameshift mutation 394delTT (a.k.a., the scandinavian mutation). 394delTT encodes a stop codon in exon 4.
- 09.07.2012
- Kalydeco (VX770/Ivacaftor)
- Have just read the devastating news on the CF Trust Forum that NICE (National Institute for Health and Clinical Excellence in the UK) are not going to recommend the National health service (NHS) provide Kalydeco in UK and want to know your thoughts and any suggestions on this to get it available? ...
- 09.07.2012
- VX770 and R347P
- Dear Team, I refer to the question you have already (partly) answered before. I think the more concrete questions is: if VX770 will help R117H - how likely is it that it can work on R347P (or any other class 4 mutation which is not a gating/class 3 mutation)? It is difficult to understand the ...
- 09.07.2012
- genetic therapy
- Hello, I wanted to have news about research on gene therapy
- 25.06.2012