Report from Europe: Ivacaftor Real World Results
http://www.cysticfibrosisnews.com/iv...m_medium=email (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 28, 2015 Category: Respiratory Medicine Authors: Imogene Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

residual function & Kalydeco news
Hi all, Does anyone has any news on the pilot study of Kalydeco and residual function mutations? On clinicaltrials.gov website it says completed and has results, but i have trouble deciphering the actual results. Maybe anyone had beteter luck? thanks (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 21, 2015 Category: Respiratory Medicine Authors: ymikhale Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

CFTR modulation for those with TWO copy's of DDF508?
OK me and my doc has talked about it and he said that i would be good for the new drug for the new gene therapy for the f508 dose anyone on here know anything about it if so please help me i cant find much online about it it kinda scars(VEST) me i have a wife and a 4 year old step son and i know what i was told was i could add years to my life witch is good but i am just scared a little bit so please help thanks so much and if you want to send it to me privet message to thanks again (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 12, 2015 Category: Respiratory Medicine Authors: Kris Salmons Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Is this the right test?
My dr ordered a genetic test it says Cystic fibrosis DNA mutation analysis with reflex to polymorphisms. Is this the appropriate test to check for milder mutations? And does it test for all the mutations? (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - May 31, 2015 Category: Respiratory Medicine Authors: Jessica Gomez Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Kalydeco with an FEV1 of ~27% (homozygous delta-F508)
Treatment with Kalydeco on patients with the homozygous delta-F508 mutation will apparently pass FDA muster in July. My girlfriend is twenty-five and her doctor has indicated that her current baseline FEV1 is around twenty-seven percent, that this treatment is intended for those with an FEV1 of thirty-five percent or higher. Can someone elucidate what he's talking about here? I fully intend to fight tooth and nail for this treatment on its approval, but I'm scared there's something I don't know that will make this impossible. (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - May 24, 2015 Category: Respiratory Medicine Authors: Bosh Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Anyone approved for Kalydeco for a 5T variant
I have a stop codon on one gene and a 5T variant on the other gene. The second gene has polymorphisms but no known disease causing mutation. My doctor is working on the insurance approval now. I have had a 6 month lung function decline, diagnosed with CFRD last week, and culture PA. Any tips or research I can give my doctor to help my cause please share. Anyone on Kalydeco with 5T variant as the only thing decreasing CFTR on that gene? (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - February 16, 2015 Category: Respiratory Medicine Authors: Beccamom Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

CFTR modulation for those with ONE copy of DDF508?
Does anyone have info on cftr modulation drugs for people that carry only one copy of the DDF508 mutation, and another of..something else? My other mutation is a nonsense mutation. Are any clinical trials going on for those of us in this boat? Or is there any research on Kalydeco combined with other drugs for the patients with one ddf508? Thanks! (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - February 6, 2015 Category: Respiratory Medicine Authors: sasha Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Z1 Auto - New sleep therapy solution, HDM
One of the medical device firm HDM (Human Design Medical) introduced a new sleep therapy solution Z1 Auto. It is going to customizes therapy for each person or patient individual by monitoring pressure requirements thousands of times per night.It also responds automatically changes in breathing patterns and delivers maximum comfort. Source: (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - January 8, 2015 Category: Respiratory Medicine Authors: emilymainzer Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Event Celebrates 25 Years of Genetic Advances in CF that Improved CF Care
Johns Hopkins University School of Medicine (JHUSOM), will be hosting a live satellite symposium celebrating the quarter century of progress that has occurred since mutations in the CFTR gene were first linked to cystic fibrosis (CF). Ahead of the Curve (AOTC): CFTR at 25 — Clinician and Patient Perspectives will take place at the 28th annual North American Cystic Fibrosis Conference (NACFC) in Atlanta on October 10, 2014. Source: http://dkbmed.com/AOTC_10-2-2014.html (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 3, 2014 Category: Respiratory Medicine Authors: jastherob Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

cystic fibrosis and criopreservation umbilical cord blood and tissue
We have a daughter of 10 months with cystic fibrosis (mutations F508del and N1303K). She was diagnosed at 5 months after 4 admissions. We saved (criopreservation) the stem cells of tissue and blood of umbilical cord of our daughter, and we still saved the umbilical cord blood of our son, older (does not carry the defective gene, he is healthy). Anyone know or can clarify me the following issues: 1. We can use the steam cells of tissue or cord blood of our daughter with healing perspectives? 2. And the brother’s umbilical cord blood? 3. Me and my husband we are 'A Rh positive' as my son, but my daughter is 'A Rh nega...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - September 29, 2014 Category: Respiratory Medicine Authors: taafom Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Mevion Higher speed pencil beam scanning - Hyperscan proton therapy system
One of the radiation therapy company from US named Mevion Medical systems introduced a Hyperscan system which is going to deliver high speed pencil beam scanning.Current pencil beam systems are very sensitive to motion.This latest Hypersacn system delivers less sensitive to patient and tumour motions.As of now it is not approved by US FDA (Food and Drug Administration). Details: (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - September 18, 2014 Category: Respiratory Medicine Authors: emilymainzer Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Medical firm BioPhotas introduced LED therapy device Celluma ELITE
Medical device firm BioPhotas announced the launch of the Celluma ELITE for sale to the aesthetic physician market.The LED therapy device is stand-mounted and able to be precisely positioned over an area of treatment via an articulating arm and a patented shape-taking light panel.The Celluma ELITE is distinct from other light therapy devices. Source: (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - September 4, 2014 Category: Respiratory Medicine Authors: emilymainzer Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Expanding Orthopedics obtains FDA clearance for FLXfit 3D expandable interbody cage
FLXfit is the world's first 3D expandable interbody cage cleared for commercialization in the United States.This unique and patented technology combines minimal invasive access, maximum footprint support and large bone graft chamber with unique lordotic expansion for sagittal balance correction. Source: (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 23, 2014 Category: Respiratory Medicine Authors: emilymainzer Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

CNBC News on Vertex...
http://www.cnbc.com/id/101783126#. (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 18, 2014 Category: Respiratory Medicine Authors: Imogene Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

New treatment for metastatic cervical cancer for women
It is good to hear that new treatment with Avastin plus chemotherapy may help women with these conditions live longer than chemotherapy alone. Recently US Food and Drug Administration has accepted sBLA (supplemental Biologics License Application).To know more details on this : http://regulatoryaffairs.pharmaceuti...160714-4318707 (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 17, 2014 Category: Respiratory Medicine Authors: emilymainzer Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

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

News for gating, residual function and conductance mutations!
Hello all, With this post I'm hoping to reach patients with gating, residual function and conductance mutations. We have found a combination of two food supplements that have a synergistic effect on CFTR and can potentiate CFTR up to therapeutic levels (around 50%). This effect is backed up by 'in vitro' and 'ex vivo' (human intestinal CF cells) research. I have posted the news on several Facebook groups last week. Some people have started trying the combination and are already noticing effects…… (including some people that are already on K) I know some of you (like ourselves) are waiting for Kalydeco to be approved soon...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 18, 2013 Category: Respiratory Medicine Authors: samaruba Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Information about how VX809 helps F508del and other mutations helped by VX809
I summarised a scientific article released in August that discusses how VX809 helps F508del, how the folding can be improved further and other mutations that were helped by VX809 in this study: http://sixtyfiverosesblog.wordpress....508del-part-2/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 18, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Nitric oxide pilot trial demonstrates an 8.8% FEV1 increase after 2 weeks
Novoteris recently announced that the FDA has granted orphan drug designation for inhaled nitric oxide as a treatment for Cystic Fibrosis. The pilot trial demonstrated an 8.8% absolute improvement from baseline after 2 weeks. Phase 2 trials are planned for 2014. More info here: http://sixtyfiverosesblog.wordpress....oxide-therapy/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 11, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

ProQR Receives Orphan Drug Status for F508del Therapy
ProQR Therapeutics recently received orphan drug designation for their Cystic Fibrosis therapy that targets the F508del mutation. This approach is different to the CFTR modulation approach (potentiators and correctors), which targets the defective protein. ProQR are targeting the Cystic Fibrosis defect at the RNA level. I have explained this further here: http://sixtyfiverosesblog.wordpress....08del-therapy/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 11, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

First Time Post
Hi! I have a 14 year old daughter who just was recently diagnosed with CF...well CRMS a few months ago and now after a hospitalization 3 weeks ago with a mucus plug and partially collapsed lung they have changed the diagnosis to CF. Her genome is M470V and r75q with a 7t/5t variance. She has a borderline sweat test. We are told this is a mild CF. Her lung functions is 75% and she is pancreatic insufficient. This is the first time I have done a forum. I woke up this morning angry, scared, and feeling very alone. My mom suggested I should join a group, so here I am. I am confused by all this genome stuff and just need some s...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - October 7, 2013 Category: Respiratory Medicine Authors: pootersmom14 Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Just lost my phase 3 place. :( DF508 /i507del
DF508/i507del (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - September 27, 2013 Category: Respiratory Medicine Authors: Rachie Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Back from Denver National Jewish.....(Kalydeco trial)
Hey Everyone! I know there were a few people interested, so I decided to start my own thread about my Denver experience. :) Sooo.... I made it to Denver on Tuesday to be screened for the Kalydeco trial. For those who don't know, it is the trial for residual function mutations. In order to get in, one must meet at least one of 3 criteria: A sweat test under 80, pancreatic sufficient, or diagnosed at or after age 12. We know that I don't meet the second two, so I can only get in if my sweat test comes in at or under 80. It was 83 when I was diagnosed in 1981, so it's really questionable. However, they seemed optimistic tha...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - September 6, 2013 Category: Respiratory Medicine Authors: triples15 Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Ataluren Update- Phase 3 trial planned for 2014
PTC had their first investor conference yesterday, here is a brief summary: - Second phase 3 Ataluren study planned to start in the first half of 2014 with CF nonsense mutations - Plan to apply to the EMA (Europe) this year - Potential for combination with Vertex's Kalydeco discussed during investor conference, PTC looking at ways to evaluate this concept More info here: http://sixtyfiverosesblog.wordpress....aluren-update/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 13, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Calista Therapeutics: F508del Research
I was just looking through my blog and realised I have not posted about Calista here. Calista Therapeutics are developing medications that target F508del. Pre clinical studies are underway and they are looking for funding for a phase 2a trial. The lead candidate is Procaftor, which appears to work to about 116% of VX809 (see the graph). I've added more info and the graph here: http://sixtyfiverosesblog.wordpress....8del-research/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 9, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Proteostasis: F508del Research
Proteostasis have identified multiple compounds that improve the folding and trafficking of F508del. These compounds have a similar level of activity to VX809, and when added to VX809, about 40% of normal function is reached. Trials are planned to start in 2015. I've added more info and some images here: http://sixtyfiverosesblog.wordpress....8del-research/ (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 7, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

809 vs 661
My husband has been thinking of getting on the phase three trial of lumacaftor plus ivacaftor. However we both have some reservations. His mutations are both delta F508. He is 28 years old. He has an FEV1 of 75%. This has been stable for the last decade. And his enzyme levels were in the normal range last time they were checked (the center still suggested he take one enzyme pill with each meal). When I read the phase 2 results for this combo, the results are dismal. There was no FEV benefit. So now for the phase III, they triple the 809 dose. This seems to be somewhat grasping at straws. Like maybe his FEV will go up 2% ...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 5, 2013 Category: Respiratory Medicine Authors: Pebbles8 Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Vertex Update: New data for other gating mutations and new trial for G551D
I've summarised the latest Vertex investor conference and added some images from the conference here: http://sixtyfiverosesblog.wordpress....vertex-update/ Main points: G551D A new study with VX661 & Kalydeco is planned for the second half of 2013 with patients who have G551D & F508del. Data is expected in late 2013 or early 2014. 2-5 year old Gating Mutations Part 2 is enrolling, results expected in the middle of 2014 Other Gating Mutations Phase 3 data: Mean absolute FEV1 improvement of 7.5% from baseline & 10.7% compared to placebo at 8 weeks (both p<0.0001). Vertex plan to apply to the FDA and E...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - August 1, 2013 Category: Respiratory Medicine Authors: GenH Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Elvated ALT levels
Hi all I have been taking Kalydeco for almost 5 months now. My liver test have been going downhill since. From normal range to an ALT level of over 250. Any words of wisdom? I have not spoken to the doc yet, but I am soooo worried. I don't want to stop Kalydeco - it is such a life changer. (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 31, 2013 Category: Respiratory Medicine Authors: sdeuber Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Australian wanting to get Kalydeco from USA or UK (or anywhere!)
Hi all. Unfortunately Kalydeco (Ivacaftor) is not yet available in Australia. Well it is, but it is $300,000 per year (well outside the price range we can afford unfortunately). We are desperately seeking a way of getting the drug from USA or UK. Does anyone know if this is possible? Has anyone done it? Can anyone work out a way it can be done? It is unbearable to know that Kalydeco is out there and can help my daughter lead a normal life, but we can't get it. Any offers of information would be most appreciated. We are prepared to do what needs to be done to get access to this life saving medication. Thank you (Source: Cys...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 27, 2013 Category: Respiratory Medicine Authors: Shani Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

Looking for anyone who has tried to get Kalydeco off label
***KALYDECO OFF LABEL*** We are trying to get together some information to help CFer's in the future get the help they need. If you feel comfortable sharing your information for the benefit of our wonderful CF community please email me at jcurrie@1starbank.com. We are looking for anyone who has tried to get on Kalydeco off label whether successful or not. We are needing your mutations and whether you were approved or denied and any information that could be helpful. Thank you in advance for any information. (Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum)
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - July 19, 2013 Category: Respiratory Medicine Authors: JENNYC Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums

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 animals), that the...
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