CFRI’s Weekly Summary of News & Events for Our CF Community!

NEW YOGA CLASS STARTS SATURDAY, MARCH 21! Yoga for CF Health and Wellbeing, a free online interactive class starting on Saturday, March 21, 9:30 am PST, is taught by Martha Modawell, Certified Yoga Instructor and mother of two children with CF. Get your mat and join your CF community members in a fun, healthy and relaxing yoga session. This class is free to individuals with CF, as well as parents, spouses, partners, and siblings of those with CF. For more information and to register, click here. Sponsored to date by Vertex Pharmaceuticals.

SUPPORT GROUP FOR ADULTS WITH CF MEETS MONDAY, FEBRUARY 17. The topic this month is "Coping with Disease Progression." The group, facilitated by Kate Yablonsky, LCSW, is open to participants nationwide, and meets from 6:00 pm PST to 7:30 pm (9:00 pm – 10:30 pm EST). To attend by phone or video call, go to https://zoom.us/j/484314120. Please log in early to make sure there are no technical problems.

CF CAREGIVERS SUPPORT GROUPS TUESDAY, FEBRUARY 18. The topic this month is "Coping with Disease Progression." The groups will be facilitated by Kate Yablonsky, LCSW. Parents of Children meet from 5:00 pm – 6:00 pm PST; to attend by phone or video, go to https://zoom.us/j/555433812. Parents/ Spouses/ Partners of Adults meet from 6:00 pm – 7:00 pm PST; to attend by phone or video, go to https://zoom.us/j/832557859. Please log in early to make sure there are no technical problems. Sponsored by Chiesi USA, Genentech, Gilead Sciences, Horizon Pharma, Vertex Pharmaceuticals, and contributions to CFRI’s CF Quality of Life Program, a Living Legacy of Peter and Kathy Judge.

REGISTRATION NOW OPEN FOR CFRI’S NATIONAL CF EDUCATION CONFERENCE! Our 33rd National CF Education Conference will take place July 31 – August 2, 2020 in Redwood City, CA. In addition to updates on new medical discoveries and therapies, you’ll have the opportunity to meet representatives from pharmaceutical and medical technology companies, as well as network with peers from the CF community. Early bird registration is $185. For more information and to register, click here. Sponsored to date by Vertex Pharmaceuticals, Genentech, Gilead Sciences, Chiesi USA, AbbVie, and Ionis Pharmaceuticals.

NOW ACCEPTING SCHOLARSHIP APPLICATIONS FOR NATIONAL CF CONFERENCE. In an effort to reduce financial barriers and expand participation among our diverse CF community, CFRI offers a limited amount of full and partial scholarships to those who cannot attend this event due to financial hardship. Priority will be given to applications received before the initial review of March 31, 2020. If there are still scholarships available after the first review, subsequent reviews are

scheduled in late April and in May. For more information and to apply, click here.

CFRI PROGRAMS AND EVENTS

TOOLS FOR COPING WITH DEPRESSION AND ANXIETY: “LIVING MINDFULLY WITH CF.” This six-week online class, starting Tuesday, March 3, draws upon Mindfulness Based Stress Reduction techniques, focusing these practices specifically to living with CF. Taught by the phenomenal Julie Desch, MD, who herself lives with CF, this class helps to reduce anxiety and depression and is offered free of charge to individuals with CF and their family members. Previous attendees are also encouraged to register. For more information or to register, click here. Sponsored to date by Vertex Pharmaceuticals, Genentech, Chiesi USA, and Horizon Therapeutics.

CFRI’S COUNSELING SUPPORT PROGRAM NOW OPEN FOR 2020 PARTICIPANTS. Children and adults with CF as well as their family members (parents, siblings, spouses/partners) are eligible to receive financial support for six individual therapy sessions per year with a licensed provider of your choice. CFRI may cover up to $120 per session for six sessions. Participants must live in the United States. For more information, click here, or email [email protected]. Made possible by grants from Vertex Pharmaceuticals, Genentech, and Chiesi USA, as well as through contributions from individual donors.

LIMITED SPACE AVAILABLE! REGISTRATION NOW OPEN FOR EMBRACE RETREAT FOR MOTHERS OF CHILDREN AND ADULTS WITH CF. CFRI’s 6th annual Embrace Mothers Retreat, with the theme "Change - Connection – Community," will take place from May 1 to May 3, 2020, at the Vallombrosa Retreat Center in Menlo Park, California. The retreat offers journaling, CF education and resources, support, art, yoga, music, and respite time with women who share the CF path. To register, click here. For more information, call 650.665.7576, or email [email protected] Sponsored to date by Vertex Pharmaceuticals, AbbVie, and Gilead Sciences.

WATCH OUR CONFERENCE PROMO VIDEO! Are you interested in learning more about CFRI’s National CF Education Conference? Are you thinking about attending but want to know what the conference is all about before you sign up? Watch our short Conference promo video here. Video made possible by Vertex Pharmaceuticals and Chiesi USA.

RESEARCH UPDATES

ORKAMBI REDUCES RISK OF HEPATIC STEATOSIS IN CF PATIENTS. A recent study has shown that individuals with CF who were treated with Orkambi have a significantly reduced risk of hepatic steatosis, a condition characterized by the accumulation of fat in the liver. Hepatic steatosis, occurring in up to 60% of CF patients, can lead to liver damage and, in extreme cases, to cirrhosis. The small-scale study is one of the first to assess the impact of CFTR modulators on CF-related liver conditions. Click here to read more.

HIGHER BLOOD LEVELS OF TINY MOLECULE MAY BE LINKED TO HOW CF AFFECTS GIRLS, STUDY SUGGESTS. Researchers discovered that girls with cystic fibrosis under the age of six have higher levels of a tiny RNA molecule, miR-885-5p, compared with boys of the same age. which could be linked to the worse clinical outcomes normally seen in women with CF. Women with CF tend to have poorer lung function, experience a higher number of exacerbations, and become infected with Pseudomonas aeruginosa earlier in the course of the disease. The presence of microRNAs in body fluids has been identified as a potential

disease biomarker, facilitating earlier and more effective intervention strategies. To read more, click here.

ADVOCACY

ICER TO RELEASE DRAFT EVIDENCE REPORT ON TRIKAFTA AND CFTR MODULATORS FEBRUARY 20th. The Institute for Clinical and Economic Review (ICER), which uses the flawed Quality-Adjusted-Life-Year (QALY) that seeks to quantify the quality and value of a life in relation to the relative value of a medication, will assess “the comparative clinical effectiveness and value of therapies for cystic fibrosis (CF),” with a focus on elexacaftor/ tezacaftor/ ivacaftor. CFRI will be sending out its response to the report upon its release, and will provide contact information for members of the CF community to share their own opinions. Stay tuned!

PATIENT GROUPS HAVE CONCERN OVER INTERNATIONAL DRUG PRICING INDEX MODEL. While high drug prices impact millions of Americans, there is concern among patient groups that the current Administration’s proposal to use the International Drug Pricing Index to lower drug prices will interfere with access to therapies. Countries with this system have worse health outcomes, as there is less access to new therapies. Canadians have little access to CFTR modulators, while in the U.K. and Europe, access is usually delayed. Click here for more information.

SUPPORT CFRI

DONATE YOUR BIRTHDAY - OR ANY OTHER SPECIAL EVENT - TO CFRI ON FACEBOOK. JOIN OUR BIRTHDAY PARTY FUNDRAISING CLUB! CFRI’s Birthday Party Fundraising is free and easy to set-up on Facebook to involve your friends in supporting CFRI. Simply go to facebook.com/cfri.org and scroll down to the “Fundraisers” section and click “Create.” To participate click here.

SHOP AMAZON SMILE AND SUPPORT CFRI! A percentage of every purchase you make on AmazonSmile goes to CFRI! It really works – we receive steady contributions, and hope they will grow. To go to Amazon Smile, click here.

OTHER PROGRAMS FOR THE CF COMMUNITY

MAKE AN IMPACT ON RARE DISEASE DAY! February 29, 2020 is Rare Disease Day! Rare Disease Day is all about raising awareness and creating change for those impacted by rare disease. The National Organization for Rare Diseases (NORD) has many events planned across the country leading up to and on Rare Disease Day. Click here for ways you can get involved and show your solidarity with the rare disease community!

THE ASSISTANCE FUND PROVIDES FINANCIAL SUPPORT FOR HEALTH- RELATED EXPENSES – INCLUDING CF. The Assistance Fund helps patients facing high medical out-of-pocket costs by providing financial assistance for copayments, deductibles and other health-related expenses. Their mission is to provide critically or chronically ill individuals with access to therapies through a continuum of services and programs. To find out if you are eligible for financial assistance, call 855.845.3663, or complete an application on their website.

PATIENT ASSISTANCE PROGRAMS PROVIDE NEEDED SUPPORT. Please see the Patient Assistance Programs on our website for information on many programs from our partners, including Genentech, Vertex, AbbVie, Novartis, Chiesi USA, and RespirTech. For more information, click here.

This eNewsletter is generously sponsored to date by Vertex Pharmaceuticals, AbbVie, Genentech, and Chiesi USA.