Cystic Fibrosis CFTR Modulation and Gene Therapy Forum Cystic Fibrosis CFTR Modulation and Gene Therapy Forum RSS feedThis 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.

Kalydeco/lumacaftor for DDF508 results are in.
http://investors.vrtx.com/releasedet...leaseID=856185 (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - June 24, 2014 Category: Respiratory Medicine Authors: Sophiesmum Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Clinical Trial for kids ages 6 and up with DD508
Hi! I was wondering if anyone has heard what is going on with the clinical trial for the combo of the Kalydeco/VX-809 for kids ages 6 and up. Our hospital was chosen as one of the sites and my son's doctor had mentioned it a few months ago... I followed up with her about it today and she said it had been put on hold. Does anyone know why? Thank you! (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - June 17, 2014 Category: Respiratory Medicine Authors: mbrandazzo Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

May Ataluren Update - New phase 3 trial to start in June
I've just added an Ataluren update to my blog (relates to mutations ending in X), including investor presentation images & clinicaltrials.gov links to the upcoming Ataluren trials: http://sixtyfiverosesblog.wordpress....aluren-update/ There is also more information & images in the March update (I have not added this link here yet): http://sixtyfiverosesblog.wordpress....aluren-update/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - May 23, 2014 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Ataluren Phase 3 Trial Results
http://ir.ptcbio.com/releasedetail.cfm?ReleaseID=848453 (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - May 16, 2014 Category: Respiratory Medicine Authors: Imogene Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

May Vertex Update- VX661 adds further benefit to those on Kalydeco with G551D/F508del
I've just summarised the May 1 Vertex investor conference, includes info about Kalydeco, VX809 & VX661: http://sixtyfiverosesblog.wordpress....vertex-update/ Also, it was announced on May 1 that the trial adding VX661 to patients stable on Kalydeco (with G551D/F508del), showed further improvements in FEV1 (+4.6%) and sweat chloride (-7mmol/L). My blog post above has more info about this. In theory this means that people who are likely respond to Kalydeco (gating, residual function) who also have F508del, may see further benefits when a corrector is added. (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - May 8, 2014 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Kalydeco and D1152h
Hi all! just wondered if anyone has managed to get kalydeco off label with this mutation? And if so what are your experiences? many thanks! Gemma x (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - April 21, 2014 Category: Respiratory Medicine Authors: Sophiesmum Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

A New Drug Device for Cystic Fibrosis Patients
Activaero has entered into research collaboration with Italian pharmaceutical firm Chiesi Farmaceutici to develop an effective solution to treat patients with cystic fibrosis (CF). (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - February 28, 2014 Category: Respiratory Medicine Authors: emilymainzer Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Feb Vertex Update - Kalydeco receives FDA approval for 8 gating mutations
I've just summarised the Feb 25 Vertex investor conference, includes info about Kalydeco, VX809, VX661 and second generation correctors: http://sixtyfiverosesblog.wordpress....vertex-update/ Also, last week it was announced that Kalydeco received FDA approval for eight gating mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. http://investors.vrtx.com/releasedet...leaseID=827435 (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - February 27, 2014 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

January Vertex Update- Latest VX809, VX661 and Kalydeco with gating & R117H info
I recently wrote a blog post about the latest Vertex news (includes info from the Dec and Jan press releases & investor conferences). Since I wrote this post it has also been announced that the combination therapy VX661/Ivacaftor has been granted breakthrough FDA designation. http://sixtyfiverosesblog.wordpress.com/2014/01/29/january-vertex-update/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - February 3, 2014 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

UK Gene Therapy Trial Update
I've recently added info to my blog about the current gene therapy trial, as well as info about a future trial that involves a new approach: http://sixtyfiverosesblog.wordpress....herapy-update/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - January 30, 2014 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Does Kalydeco Eliminate Pseudemonas?
I'm just wondering. If Kalydeco goes a long way toward normalizing lung function, have users found they are finally able to rid themselves of pseudemonas or other colonized bacteria? (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - January 9, 2014 Category: Respiratory Medicine Authors: Gammaw Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Ataluren and Nonsense Mutations Update
I just added two blog posts, one about Ataluren, with information about the upcoming trial & EMA application, and the other about a new potential approach for nonsense mutations (mutations that end in X). New Potential Approach for Nonsense Mutations November Ataluren Update (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - November 26, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

3 y/o post lap chole
Just checking how many have dealt with their kiddos having a cholecystectomy?? My 3 y/o son had his gall bladder removed at the end if October after having many issues eating. Biopsy showed chronic inflammation. Surgeon said this was common with CF kiddos. since surgery he is having some biliary spasms and severe back pain intermittently. His appetite is better overall, but wondering how long this might go on. He is on Levsin to help with the pain. Just checking to see how everyone's else's kids faired afterwards. (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - November 23, 2013 Category: Respiratory Medicine Authors: tysmama Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Surfing-inspired nebuliser developed to treat cystic fibrosis
Benefits of saltwater treatment for CF. Here are full details: http://anesthesiarespiratorydevices....ibrosis-240513 (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - November 11, 2013 Category: Respiratory Medicine Authors: emilymainzer Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Australian Kalydeco Campaign - New Facebook Page
Kalydeco is still not available to the 200 Australians with G551D. The reimbursement has been delayed as meetings and negotiations continue between Vertex and the Australian PBAC committee. We have a campaign through facebook and twitter, with the new YesToKalydeco page https://www.facebook.com/Yestokalydeco and our twitter account https://twitter.com/KalydecoAus. We would really appreciate if you could like/share our facebook page and follow on twitter. It looks likely that August 2014 is about the minimum timeframe for availability, so we need as many supporters as possible to try to accelerate this timeframe! Thank...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - November 8, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums