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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 brothers 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