Here you will find the most recent organizational news from FARA, including information on events, and awareness and advocacy initiatives. To locate an article from a certain date, please use the archives on the right side of your screen.
Student battling rare disorder — with a smile — wins national writing contest
A Grosse Pointe fourth-grader's experience with a rare disease inspired her to write a letter that won first place in a national writing contest.
And Claire Juip, 9, is herself an inspiration — to her family, to her friends and to the community around her school, St. Paul Catholic School in Grosse Pointe Farms.
She and her brother, 11-year-old Jake, have Friedreich's ataxia. The genetic condition causes progressive damage to the parts of the nervous system involved in muscle control.
Despite the prospect of likely having to use a wheelchair someday, both children are filled with optimism, working to educate people about the condition and participating in sometimes painful medical studies in the hope of helping others.
Four National Organizations, Hundreds of Families Impacted, Join Together to Speak with FDA about Drug Development in Friedreich’s Ataxia
HYATTSVILLE, Md. June 1, 2017 — The Friedreich’s Ataxia Research Alliance (FARA), National Ataxia Foundation (NAF), Muscular Dystrophy Association (MDA) and Cure FA Foundation will host the first ever Friedreich’s Ataxia (FA) Patient-Focused Drug Development (PFDD) meeting on Friday, June 2, to give patients, families and caregivers the opportunity to speak directly to the U.S. Food and Drug Administration (FDA) about the impact FA has on their daily lives, and their highly unmet need for effective treatments.
FARA to Ring The Nasdaq Stock Market Closing Bell - Friday, May 19th
The Friedreich’s Ataxia Research Alliance (FARA), a non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich's ataxia will visit the Nasdaq MarketSite in Times Square for Friedreich’s Ataxia Awareness Day.
In honor of the occasion, Ron Bartek, President and Co-Founder and Paul Avery, Chairman of the Board, will ring the Closing Bell.
JOIN AN UPCOMING LIVE WEBINAR TO LEARN ABOUT THE PATIENT FOCUSED DRUG DEVELOPMENT MEETING
JOIN AN UPCOMING LIVE WEBINAR on April 4th or April 11th TO LEARN ABOUT HOW THE FA COMMUNITY CAN PARTICIPATE IN THE PATIENT FOCUSED DRUG DEVELOPMENT MEETING WITH THE FOOD AND DRUG ADMINISTRATION
The Friedreich’s Ataxia Research Alliance (FARA), in partnership with the Muscular Dystrophy Association and the National Ataxia Foundation, will be hosting two live webinars in April to discuss how FA patients and caregivers can participate in the very important, upcoming Patient Focused Drug Development meeting with the U.S. Food and Drug Administration (FDA) in June.
Please join us for one of the two live webinars (click "Read More" below) to view key information, including background information about the FDA & drug development, an overview of FDA Patient-Focused Drug Development, discussion questions & format, tips for participating in the meeting, and meeting logistics.
Both webinars will cover the same material. To dial in, click on the appropriate link above and follow the instructions (hit the “More information” button for the meeting # & audio #). Plan to dial in five minutes early if you have never used WebEx (meeting software), as you may need to download software before joining. Participation in each webinar is limited to the first 100 people who join. The webinars will be recorded and can be viewed later for those who cannot participate live.
Our goal is to have as many patients and caregivers as possible participate in the Patient Focused Drug Development meeting (either in person or via live webcast). This meeting is your opportunity to tell FDA and drug developers about the challenges and burdens you have experienced with FA, and to share your thoughts about what is most important to you in evaluating potential new treatments for the disease. This meeting marks the first-time patients and their families affected by FA, and other caregivers, will be able to speak directly to the FDA and share their experiences in their own words.
Should I participate in a webinar if I cannot attend the FDA meeting in person?
YES! Please join us for one of the upcoming webinars to learn about the FDA meeting because even if you cannot attend in-person, we would like you to participate online via a live webcast. You will be able to actively contribute to the meeting and share your input on the specific panel questions, as well as demographic questions with the use of your mobile device (via texting) or via a web page.
FDA Meeting Date & Location
Meeting location: College Park Marriott and Conference Center, Hyattsville, MD Date & Time: June 2, 2017, 8:00am - 12:30pm
* Following the FDA meeting, there will be a research update for anyone who is interested (1:30-3:00 pm)
Rare Disease Week on Capital Hill, February 27 - March 2, 2017
Ron Bartek and Brigid Brennan joined rare disease advocates from 48 states for the annual Rare Disease Week in Washington DC. The theme this year was research - a mission FARA has been supporting for almost 20 years. Here are some highlights:
Monday - Rare Disease Day at the National Institute of Health (NIH)
It was standing room only as doctors and advocates shared updates from their respective fields. Over 700 people were present and, for the first time, many more livestreamed from home. Everyone was in agreement that research is needed in the rare disease community with less than 500 treatments for over 7000 diseases. However, we are not alone in benefitting from goal. Rare disease are often the "window" into common disease treatments as well. Rep Leonard Lance (R-NJ) spoke about his continuing support for the rare disease community. The crowded erupted in applause to learn that Dr Francis Collins, a champion for our community, was asked to stay on as head of the NIH.
That evening Rare Disease Legislative Advocates (RDLA) hosted a cocktail reception and documentary screening of Up for Air. Senator Ed Markey (D-MA) and Representatives Jim McGovern (D-MA) made brief remarks of support. Our own Ron Bartek moderated the panel discussion of the movie where everyone left in awe by Jerry Cahill's inspirational journey with Cystic Fibrosis.
Tuesday - RDLA Legislative Conference
Approximately 350 advocates gathered to learn a wealth of information on the new Congress, The Trump administration, top health policy issues and working with federal agencies. Most importantly, the experts shared tips on how to effectively meet with your legislator and continue building that relationship after you have left. With so many key pieces of health care legislation currently in debate, there has never been a better time for all of us to engage with our representatives. This conference was also livestreamed for the first time but if you missed it, video and presentations will be available on the Legislative Conference resource page. www.rareadvocates.org/207-legislative-conference-resources/
Wednesday - Lobby Day
The day started with the opportunity to gain some legislative insight from Representative Gus Bilirakis (R-FL) and former Representative Brian Baird (D-WA) over breakfast. Then Dr Janet Woodcock, Director of the Food and Drug Administration's Center for Drug Evaluation and Research, addressed the crowd. A resounding note of appreciation was heard in the room for Dr Woodcock's continued support in incorporating the patient perspective in research.
Then we were off to the hill! 328 advocates participated in 270 legislative meetings. Everyone was grouped by state and meetings ranged from 1 to over 20 people. The asks this year included:
· The Open Act - which would support repurposing already approved medication · Affordable Care Act (ACA) - provides important coverage for the rare disease community including no pre-existing bans, no lifetime limits, coverage until 26 · Funding for NIH/FDA - without appropriate funding, neither agency can hire the needed doctors and scientists to maximize research potential · Rare Disease Congressional Caucus Most legislators were supportive of our general concerns and requests. However, with so much uncertainty on the hill around health care and funding, it is important for us to stay firm and persistent on the issues that matter the most to us.
A long but successful day was wrapped up with RDLA hosting a Rare Artist Reception in which advocates had another opportunity to talk to attending legislators and staff members while everyone enjoyed seeing the winning art entries from the rare disease community.
Caucus co-chair Senator Amy Klobuchar (D-MN) welcomed the advocates and Congressional staff to the final event of the week. A review of the 21st Century Cures Act, the PDUFA Reauthorization Process as well as the ACA were reviewed. Video of the meeting will be posted on the Caucus webpage within a few weeks. www.rareadvocates.org/rare caucus/
The week provided a great resource for information but also a great opportunity to share strategies and form coalitions. Together our voices were a roar in DC.
Written by Brigid T Brennan, Esq Director/In-House Counsel/Advocacy