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Hi bookworm, I did get your form submission feedback in my inbox and will take a look at this thumbs issue. Thank you for pointing it out to me!
- Chris
Thanks to generous donations from the Boler and Parseghian families, the University of Notre Dames's Center for Rare and Neglected Diseases is renamed to the Boler-Parseghian Center for Rare & Neglected Diseases. A donation of $10 million dollars to the center will go towards funding further...
Vertex trial results recently presented at the NACFC, spurred several stock analysts to increase expectations for the company’s shares. 22 out of 34 analysts covering Vertex now have a positive rating on the stock.
Vertex recently provided data showing its experimental drug, lumacaftor, and its...
Hi Littlemyth, what a nice video you have made for little Jude! I really enjoyed it and shared it on the CysticFibrosis.com Twitter account https://twitter.com/KnowCF ... every bit counts so keep up your efforts!
N8 Respiratory presented data at the NACFC demonstrating antimicrobial efficacy of CSA-13 against Psuedomonas aeruginosa (PA) biofilms. Specifically, a preclinical study was presented demonstrating its lead compound CSA-13's efficacy as an antimicrobial peptide mimic against Pseudomonas...
Jennifer Doudna and her colleagues found an enzyme in bacteria that makes editing DNA in animal cells much easier. For this discovery, Jennifer Doudna maybe in line to win a Nobel Prize in the coming decade.
Until now, the tools for fixing or replacing a gene in animals were cumbersome, if...
Hi Roxanne, Thank you for starting off this new forum with this excellent initiative! I have liked your facebook page both personally ( https://www.facebook.com/cysticfibrosisdotcom ) and with the official CF page ( https://www.facebook.com/knowcf ) and twitted you fundraiser out to our Twitter...
Hi Kim, you have my vote! Thank you for bringing this to my attention by posting at the CysticFibrosis.com Community forums. I really hope Faith is doing well and will get the (free) roof she so desperately needs to stay healthy and away from dangerous infections. This reply should also...
Here are the highlights from Vertex's CF R&D programs presented at the NACFC.
1. Interim analysis of rollover study following the Phase 3 TRAFFIC and TRANSPORT studies showed sustained improvements in lung function through 48 weeks of treatment with lumacaftor in combination with ivacaftor in...
Galapagos, a Belgium biotechnology company recently presented novel corrector / potentiator combination drug candidates for restoring CFTR function at the NACFC. Pre-clinical data was shown in which these novel corrector molecules in combination with existing CFTR modulators restored at least...
Use of Kalydeco in Children Ages 2 to 5: Vertex today announced the submission of an NDA in the United States for the approval of Kalydeco in children with CF ages 2 to 5 who have one of the following nine mutations in the CFTR gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P...
A very interesting research topic will be presented at the upcoming NACFC where scientists report a novel approach to treating CF lung disease. They claim that a polymer (called PAAG) which is based on a natural polysaccharide, is able to breakdown unwanted mucus and bacterial biofilms. The...
Hi Sara, welcome to the forums! This is a great place to connect and learn from others that are going through what you are going through. I am sure there are many here that can help you. Califormia is a beautiful place and the fresh salty air from the pacific ocean should help a lot! Will you be...
Maybe we'll have CF Bots in the future...they'll take care of all your pills, presenting them at the right time in the right amounts. They'll take care of all the devices, clean them order new parts when they break. They'll take care of reminders and appointments with doctors and therapists...
Drug combination therapies to treat the underlying cause of Cystic Fibrosis are a promising strategy and here is why: Cystic Fibrosis is a result of specific mutations that occurs in the CFTR gene, resulting in a defect CFTR protein. The combination therapies currently being tested are...
Data Driven Medicine is leading us to personalized medicine. Advances in Next Generation Sequencing (NGS) and High Performance Computing (HPC) are enabling P4 medicine: personalized, predictive, preventive and participatory. This new era of medicine ensures every patient is well diagnosed and...
A drug currently in phase 2 clinical trials for COPD and Asthma maybe effective for the treatment of Cystic Fibrosis. Verona Pharma is expected to present some data at the upcoming NACFC on their drug called RPL554, which they claim will help "activate" the CFTR protein. CFTR activation may also...
Someone asked me to explain the difference between mucoid and non-mucoid Pseudomonas aeruginosa (PA) and how this relates to Biofilms. The answer is quite simple really. Most people with CF become infected with the non-mucoid PA strain in their childhood or adolescent years (80% are infected by...
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