Cystic Fibrosis CFTR Modulation and Gene Therapy Forum This is an RSS file. You can use it to subscribe to this data in your favourite RSS reader or to display this data on your own website or blog.

V520f
I am desperate to know any others with the V520F mutation, or if anyone knows what class of mutation it is. The little bit of information I can find on it makes no sense to me. I'm just wanting to know if I can fight for Kalydeco for my daughter with it. Her other mutation is Delta F508. Thanks so much, Amy (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 17, 2013 Category: Respiratory Medicine Authors: amykay1965 Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Today show and Kalydeco
I can't believe that no one has posted this yet!!! If you haven't seen this today....you need to take a minute and watch it!! This is a story about how siblings that have the same Dr, same CF genes, and same health insurance got two totally different responses to their Kalydeco request!! http://www.today.com/news/man-battles-health-insurer-drug-could-save-his-life-6C10636133 This is an amazing story!!! I guess we were on the right end of the russian roulette!! Scary!!! (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 16, 2013 Category: Respiratory Medicine Authors: JENNYC Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

New Drugs and insulin use
Hi all. I read the thread about decrease in need for enzymes since starting the new drugs but I was wondering about insulin. Has anyone with diabetes seen a reduction in their insulin use/need? Hoping to get in on the phase 3 trial as I am double delta F508. Thanks. Kalen (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 10, 2013 Category: Respiratory Medicine Authors: ksy8384 Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Abby Clinic Update
I have updated everyone on facebook but I haven't been on here in quite some time because I have been on my first Vacation since Abby was diagnosed. I actually got to take my full 2 weeks and didn't have to spend my entire vacation at the hospital!!! It was fantastic!!! It would have been better had we have not been broke but still loving life with Kalydeco!!! So Abby has been taking Kalydeco since June 28, 2012. We went to clinic a couple of weeks ago and discovered that we had a completely new CF team! Dr was gone...fellow leaving...and nurse had a baby and not coming back. Wow!! So here is the comparison: ........
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 9, 2013 Category: Respiratory Medicine Authors: JENNYC Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Ataluren Update
A recent study found that Ataluren failed to enhance read through past stop codons (they tested all the possible stop codon combinations in different situations), instead they suggested that Ataluren interacted with the drug screening system, the luciferase enzyme. I've summarised the findings here: http://sixtyfiverosesblog.wordpress....aluren-update/ “Where does this leave us with PTC124 and its potential to tackle the 10% of cases of genetic disease that are thought to result from nonsense mutations? We should remember at this point that McElroy and colleagues only test cells (not intact animal...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - June 26, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums