The latest video in The Path Forward with Cystic Fibrosis series, Dr. Emily DiMango, Director of the Gunnar Esiason Adult CF Program at Columbia University Medical Center, discusses the lung transplant process through the lens of a CF doctor.
First, she reviews the importance of CF patients participating in drug trials in order to start life-changing medications sooner. She then answers the following questions:
· What does pre-transplant management look like for a CF patient?
· When is the right time to be referred to the list?
· What is the referral process like?
Finally, she reiterates the importance of well-rounded treatment that includes physical health, nutritional health, and emotional health.
This video was originally posted on JerryCahill.com
Cystic fibrosis is a devastating disease. About 30,000 children and adults have cystic fibrosis (CF) in The United States. While the life expectancy for someone with CF has doubled in the last 30 years, children and adults people still lose their lives to this disease every day. There is no cure yet. It’s time to find it.
How can you help? Use your voice and get involved! Participate in Great Strides walks and other events supporting CF and share the facts on social media and in your community. The Cystic Fibrosis Foundation is working hard to find a cure. Other foundations and organizations help the CF community as well. Like the CF Roundtable which supports adults with CF, there is a foundation that supports children and families living with CF: Claire’s Place Foundation.
Claire’s Place Foundation, founded by Claire Wineland, when she was just 14 years old, is a 501c3 non-profit organization designed to help children and families affected by cystic fibrosis. Claire was born with CF. In her short life, Claire has undergone more than 20 surgeries and has spent months at a time in the hospital. She survived months in a coma on mechanical ventilation despite the odds and was also a Make-a-Wish recipient. She has 40% of ideal lung function and the doctors have estimated two years left to live. This is not, however, what characterizes or defines her. Claire has dedicated her life work to making a difference in the lives of others living with CF.
Now 21 years old as an adult living with CF, Claire’s Place Foundation is a way for Claire to give back with hope, strength, and joy. She wants to live with intention and purpose. In her own words, “I know my life will be shorter so every day has meaning.” She strives to find new ways to help the CF community and other children afflicted with chronic illness. Claire’s Place is making a huge impact through its two programs – The Extended Hospital Stay Fund and the Family Support Program. These programs are in high demand and the foundation needs your donations to continue assisting young patients in need. You can meet Claire and support her foundation at her next fundraiser, “Glow Ride,” on August 18, 2018 in Hermosa Beach, CA.
To find others ways you can help bring awareness to CF find a local chapter near you.
Get involved. Donate. Come to events.
An interview by: Clara Rodríguez Fernández
Daniel de Boer founded ProQR in 2012 following a strong determination to improve the lives of people with cystic fibrosis. “We started ProQR Therapeutics for a very personal reason,” he told me. “Eight years ago, my son was born, and diagnosed with cystic fibrosis. At that time, I was a serial entrepreneur in IT. I decided to make a career switch and start a company to develop drugs for cystic fibrosis, but then also for other genetic diseases.”
One would think that a person without a background in biotech might have it difficult to succeed, but de Boer is not the only to have so far successfully undertaken this endeavor. Over in France Karen Aiach built Lysogene to treat her daughter’s rare genetic disorder, while in the US the story of John Crawley and his company Amicus Therapeutics, founded to help his two children’s diagnosis, went so far as to inspire a movie. The determination and motivation of these parents seem to overdrive any challenges they might have faced because of their limited experience.
De Boer set out to create a business plan for his new company and found out that there was already quite a lot of activity, especially in approaches using small molecules or gene therapy.“We decided that we really wanted to add something new to the space, and take a completely novel approach.”
So he started looking for a new technology, and he found it. “Around that time, I met for the first time with some people in biotech, including the CEO at Alnylam, John Maraganore, and we talked about how they used RNA approaches for genetic diseases,” says de Boer.
Technologies targeting RNA are quite new compared to those that target DNA such as gene therapy. But RNA-based treatments have started to gain traction in the last few years. There are multiple ways that RNA can be used as a therapeutic, but its distinctive advantage over gene therapies and the likes is that it does not permanently change our genetic makeup, making it possible to reverse its effects.
Today, RNA technology is being tested in multiple rare diseases caused by genetic mutations, such as hemophilia, porphyria, or iron overload disorders. “I thought, ‘if you can do that for all these other genetic diseases, why not for cystic fibrosis?’” says the Boer. “With that in mind, we started ProQR.”
For the rest of this article, please click here.
We would like to thank The Boomer Esiason Foundation for its continued support at the Pearl Sustaining Partner level. A special thank you goes out to BEF volunteer Jerry Cahill for helping make this grant possible. Because of this support, we can provide all of our CF Roundtable programs such as:
- CF Roundtable Newsletter
- CF Roundtable Blog: https://goo.gl/iqsNhc
- The Speakers Bureau: https://goo.gl/WNdtDX
- Awards to individuals who make a difference in the CF community: https://goo.gl/6LfGXG
- Support to college students tackling CF and an academic schedule: https://goo.gl/AvksXN
The Boomer Esiason Foundation helps support the CF community via its programs including:
- Scholarships – BEF has numerous scholarship opportunities available
- Lung Transplant Grant Program – covers transportation, housing and other expenses not covered by insurance that are related to transplant
- You Cannot Fail – A motivation program that empowers people with CF
- CF Podcasts – podcasts covering a wide variety of CF-related topics produced by Jerry Cahill
- CF Wind Sprints – short videos by BEF Volunteer Jerry Cahill with tips for living with CF
- Gunnar’s Blog – a personal blog of Gunnar Esiason, Boomer’s son, who has CF
- Hospital Bags – goodie bags provided to CF patients of all ages during hospital stays
- Team Boomer – encourages people with CF to be active by participating in events and helping to fundraise
- Bike 2 Breathe – An annual 500-bike tour to raise awareness for the importance of exercise with CF
- CF Century Rides – A personal goal of Jerry Cahill’s. Jerry is determined to do a century ride (100 miles bike ride) in all 50 states for CF!
- CF: Live By Example – A pilot program where people with CF who are living, breathing, and succeeding will ensure parents of newly diagnosed children that CF is only a bump in the road, not a death sentence.
- Club CF – an online forum where people with CF can share their stories
For more information on The Boomer Esiason Foundation please visit: https://www.esiason.org/
Hi! My name is Rachel Steinman, I’m 29 years old, and I’m super excited to be a member of the USACFA board.
I was diagnosed with CF at the age of 16 and have been very fortunate to have had fairly stable health throughout my life thus far.
No matter one’s level of health, USACFA is an important outlet for our community. CF is a complicated disease and it affects every patient in a unique way. For me, having an online community has helped me both learn more about this disease, and feel less isolated in the process.
I graduated from the University of Miami with degrees in Journalism and Sociology in 2009, and I spent a year volunteering with Americorps shortly after. A few years into my career I decided to quit my job and move to Tel Aviv, Israel for a change of lifestyle and a marketing position with a tech company. I moved back home to NY after a year to be close to my family.
I grew up on Long Island and currently reside in NYC with my boyfriend where I continue to work in digital marketing. I enjoy cooking, traveling with my boyfriend, and spending time with friends and family.
I believe I’ve been able to maintain good health with the help of a great team of doctors at Columbia Presbyterian in Manhattan, a positive attitude towards life with CF, lots of acupuncture and cupping therapy, and a very loving, supportive family.
As a new member of the board, I’m excited to be joining both the blog and social media committees, so please look out for future posts from me!
Hello! My name is Amy Sylvis and I am so grateful to be a member of the USACFA board. I have been an avid reader of the CF Roundtable since the late 90s and I am forever grateful for how much I have learned over the past 2 decades from the publication. I fiercely believe that all people with cystic fibrosis should have access to the best care and latest knowledge – and I’m thrilled to be able to contribute to this prestigious organization. My specific passions include hemoptysis, aspergillus, CFRD and decreasing quality of treatment variation across CF centers in the United States.
I am 36 years old, diagnosed at 6 months old. I earned my Bachelors of Science in Business and Bachelors of Arts in International Relations from the University of Southern California as well as my Masters in Business Administration from USC. I have worked full time mostly in biotech and pharma, which continues to be my passion despite my health forcing me to leave work in 2017. In my spare time you can find me reading non-fiction, watching college football and traveling. My husband and I were married in August 2017, and we live in Los Angeles, CA with our little cocker spaniel/dachshund mix.
The Case for Realistic Optimism
Have you ever struggled to stay positive when dealing with a sick loved one? When Ray’s wife Rebecca went into respiratory failure from end stage cystic fibrosis he was faced with this challenge. What he learned was that choosing to be realistically optimistic helped him to remain strong for Rebecca during Continue reading TEDx talk: The Case for Realistic Optimism
PMD Healthcare, a medical device, digital health and chronic care management services company, has announced it is forming two new patient advisory boards: one for Cystic Fibrosis (CF) patients and caregivers of CF Children, and one for Pulmonary Fibrosis (PF) patients and PF caregivers.
By: Laura Mentch
Our story begins with Lisa McDonough, a young woman with CF. Lisa wanted to find a way for people with CF to connect with each other and share their thoughts and strategies related to living with CF. In 1989 she singlehandedly produced 4 issues of Roundtable, a newsletter for adults with CF. Lisa knew that she benefited from talking to people with CF on the phone or in CF clinic and she wanted other adults with CF to benefit from connecting and sharing information about living with CF, medications, and staying healthy. Continue reading USACFA History: How a group of adults with CF helped create a community without computers, email, or the internet
The US Adult CF Association (USACFA) is excited to announce our recipients of the Lauren Melissa Kelly Scholarship for the Spring of 2018.
In our evaluation, we look for students who demonstrate tremendous academic achievement, community involvement and a powerful understanding of how having CF matched with these achievements places them in a unique situation to gain leadership roles within the community. Our scholarship is open to all pursuing any degree, from associates to Ph.Ds. We believe that any higher education is a strong foundation for advocacy and involvement in CF.
We are pleased to announce Hannah Buck and Mary Grace Bernard as the recipients of this semesters’ scholarship. Congratulations to them! They will be awarded $2500 each.
Both of our recipients demonstrated the leadership, intelligence, and drive of Lauren Melissa Kelly. We at USACFA look forward to seeing them further develop their leadership and advocacy in the cystic fibrosis community.
We are also pleased to award runner-up scholarships in the amount of $250 to five deserving applicants: Elizabeth Shea, Chloe Creager, Rebekah Weigner, Holly Beasley and Christopher Davis, Jr.
We are excited to announce more scholarship opportunities coming soon! Please stay tuned for more information. For questions, please contact us at firstname.lastname@example.org.