Lucky CF Mom | Possibilities
51 FOLLOWERS
I have the most amazing good luck. 8 years ago, my world came crashing down when my newborn baby was diagnosed with the genetic disease cystic fibrosis. Today, we feel like the most fortunate family in the world because we have Kalydeco! Now my goal is to work toward more personalized research, to help bring the most effective treatment to each and every person living with CF!
Lucky CF Mom | Possibilities
3y ago
Every year at the NACFC, one symposium session is dedicated to debating hot topics in CF clinical care. This year's symposium addressed three important issues, where specialists have significant differences in opinion. One expert presents the PRO argument, another presents the CON argument, and then the audience "votes" for the winner by applause.
This year, 3 important issues were debated--
1) Can Exercise Substitute for Airway Clearance?
2) Which should be the first drug introduced to manage CF--Hypertonic Saline or Dornase Alfa (Pulmozyme)?
3) Should glucose intolerance be treated ..read more
Lucky CF Mom | Possibilities
3y ago
The Conference may be over, but I am going to keep the information coming! I am trying to overcome the feeling that I need to sit down and write this huge novel for a proper blog post...so this entry will focus on information from a single discussion I watched at the NACFC entitled: The Developing Gut and Respiratory Tract Microbiome in Infants with CF.
The term "microbiome" refers to the community of microorganisms that live in our guts and lungs. Healthy biomes are generally non-pathogenic (not disease causing)--they exist in harmony with our bodies. Problems begin to o ..read more
Lucky CF Mom | Possibilities
3y ago
The third Plenary Session of NACFC 2013 explored the topic of Cystic Fibrosis Related Diabetes (CFRD) and was entitled: CFRD: From Bench to Bedside & Back again. Andrea Kelly, M.D., MSCE was the first speaker in this session.
Patients with CF are living longer than ever before (YES!), but age can bring new health issues--including CFRD. CFRD is very common--50% of CF patients will develop it by age 30. Current guidelines recommend annual oral glucose tolerance tests beginning at age 10.
Insulin secretion defects are present early in CF patients, and are progressive. We ..read more
Lucky CF Mom | Possibilities
3y ago
Today started out with the second Plenary session: Roadmap to a Cure: Part II. The discussion was given by Dr. Bonnie Ramsey, University of WA School of Medicine, Seattle WA. Dr. Ramsey is also the Director of the CFF Therapeutics Development Network. She is extremely smart and we are all lucky she has dedicated her career to CF.
Dr. Ramsey’s talk went into detail about how the CFF is working to “leave no mutation behind.” She talked about what was learned in the process of getting a successful FDA approval for Kalydeco and how we can use that “roadmap” to gu ..read more
Lucky CF Mom | Possibilities
3y ago
Gene editing and mRNA editing have been discussed at NACFC for a few years now. Here is the link to last year's blog and conference slides. Researchers have been working hard to overcome obstacles to delivery and expression of genetic treatments over the course of the last year...and they are getting somewhere. I spent Friday afternoon, as well as Saturday morning listening to talks about gene editing. Check out this animation illustrating what gene editing might look like in cystic fibrosis.
As seen in the video, nucleases can be engineered to create site specific, double strand breaks at ..read more
Lucky CF Mom | Possibilities
3y ago
This is my 7th year attending the NACFC...and after 3 days of Workshops, Symposia, and Plenary Sessions--I am more optimistic than ever. At the NACFC, I try to take every opportunity I can to soak in the science and connect with the researchers. This year was the largest NACFC ever--with close to 5000 in attendance from all over the world. I skip the gym, sleep, and regular meals to that no opportunities escape me. This entry will touch on exciting news from the first Plenary Session.
2020 is going to be a BIG year for treating Cystic Fibrosis
The first Plenary Session always features ..read more
Lucky CF Mom | Possibilities
3y ago
Ironically--fighting for healthcare may stress me to death. I'm struggling to stay positive, but it helps to know I'm not alone. My Mama Bear Defense System is humming at full throttle. I'm anxious, and frequently on the edge of tears... I'd love to just take a vacation and check out, but now is not the time to take our eyes off the ball in terms of advocating for healthcare policy for those with cystic fibrosis.
The CF community engaged with lawmakers in a formidable way in opposition to the GOP's American Health Care Act (AHCA), providing feedback about the inadequacies of the legislation w ..read more
Lucky CF Mom | Possibilities
3y ago
As National Advocacy Co-Chair for the Cystic Fibrosis Foundation (with my fabulous Co-Chair Melissa Shiffman), and Mother to a 9 year old living with cystic fibrosis, my level of anxiety has been elevated to some all new heights as the dismantling of our healthcare policy looms on the horizon. A second contributor to my overall mental health crisis is the INCREDIBLE LACK OF DETAIL about what will be proposed to replace the ACA. By all sources, the 1st order of business for the incoming administration will be the repeal of large portions of the Affordable Care Act ("Obamacare"). The ACA i ..read more
Lucky CF Mom | Possibilities
3y ago
From my experience, the NACFC has always been saturated with heavy science…which is fantastic, and has helped create what many may call the most incredible story of progress in medicine today. I attended my very first Conference in 2011 to see the science for myself, and hell would have to freeze over to keep me away at this point. In my 6 years attending this event, I’ve watched the CF research environment EXPLODE. There are 50+ clinical trials that will take place in the coming year, and more companies are entering the ring of CF drug development than ever before. The NACFC ..read more
Lucky CF Mom | Possibilities
3y ago
Dr. Michael Boyle wrote a fantastic narrative of the second Plenary session. Check out the corresponding slides below! My favorite part of this session was the live involvement of 2 adults living with CF, and the Mother of twins with CF! Bringing the patient voice front and center at a research focused Conference is truly groundbreaking! Enjoy ..read more