Living in Athens with CF Recommended ?
I have an 11 year old daughter with CF and we live in Abu Dhabi, UAE, but we may come to live in Athens, I am worried about the air pollution, we will be living in the hills outside Athens. Also Gala has just started to take Ivacaftor as she has the G551 mutation, is this going to be available ...
Welcome 00
Are Ataluren the benefit of all cases of muscular dystrophy or limited to only Duchenne and Becker? If so is there another drug handling other cases of muscular dystrophy? I hope so Heard that Ataluren is perhaps approval from before inguinal Union in the beginning of the year 2014? Is this ...
Muscular dystrophy
What is the latest news about drug PTC124 (Ataluren) ? and Does medication proved its effectiveness in the treatment of muscular dystrophy?  I heard that the drug will be available at the end of 2013 or the beginning of 2014 Is this true? I do not know if it has been the answer to this ...
D1152H and G85E
Hello, my son (5 months plus) has inherited the D1152H and G85E mutations. His sweat test at 14 days old came back borderline at 33. His first two fecal elastase tests came back borderline, but his most recent test came back normal. Overall he has been healthy thus far. Any info on these two ...
cistic fibroses
DEAR EXPERT TEAM i would like to know more about the availabilty of Kalydeco in Bulgaria and if there will be any point for someone suffering from CF with mutation 621+1G/T,N1303K to try this medication.and also if not suitable what other treatment should we look for in order to keep quality of ...
Two Class I mutations
Hi our son has two class one mutations, 3659delC and 2183AA>G. My research has shown that these are both frame shift mutations. Are there any drugs or studies for people with two Class I mutations?
VX Combo trial 770/809
Hello, how long will the combo trial phase III last? When will the results be presented? Thank you, Best regards,
Test question for keyword list-please ignore
This is a test question, please just ignore it
my sun 10 ag has duchin musquler dystrophyl please haw can I get the ptc 124
394delTT and Ataluren
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 axon 4. Is it possible that Ataluren (by supressing the stop codon) could enable ...