Your Community News
Hello friends,

As you know, May is FA Awareness month. A special thanks to everyone who got involved in the campaigns, events and talks throughout the month. Members of the community flexed (at home) to raise funds through the social campaign #LendUsSomeMuscle, logged miles and fundraising dollars in the rideATAXIA NorCal Virtual Challenge and the Gehr Family hosted a live stream of awesome musical performances in this year's Cure FA Soirée. Pharma partners Exicure, PTC Therapeutics and Avexis all honored FA Awareness month by inviting FARA and FA patient panelists to their virtual team meetings. And FARA hosted our own flash-talk series featuring presentations from Young Investigators on their FA research.

There is always a way to remain active, even from home. One specific research need is for Parents of FA Children age 12 and under and Patients ages 13-17 to complete The Friedreich's Ataxia Health Index Study. This study aims to identify the symptoms that have the greatest impact on quality-of-life for individuals with FA. The results of the study will help guide future research involving FA patients through the development of a patient-reported measure of health. The study involves completing a survey to include questions on demographics and symptoms of FA. Please see this recruitment flyer for more information and to access the survey links. 150 responses each from Parents and Patients are needed to validate the study outcomes. Thank you for all you continue to do to advance FA research.


Jen Farmer,
Chief Executive Officer
Open & Enrolling Studies
During this time of social-distancing, let's keep the momentum going for FA research. The studies below are recruiting by phone screening, until travel to clinical sites can resume.
IDEA Study
FARA is supporting the IDEA research study to test body-worn sensors to measure movement. The goal of the study is to evaluate progression and severity of ataxia. The study involves visits every 6 months over 2 years at a clinic located in Baltimore, Boston, Chicago, or Los Angeles. Participants must be 12-30 years of age and be able to walk 10-feet independently, without an assistive device. For additional eligibility criteria and clinics open for enrollment, please contact study coordinator Hannah Casey ( ) or (773) 702-4610. 
Retrotope RT001-006 Phase 2/3 trial for protection against oxidative damage in the central nervous system
Retrotope is seeking 45 volunteers to participate in a clinical trial for the drug RT001, which may protect against lipid peroxidation, the process that is believed to cause disability in many neurodegenerative diseases, including Friedreich’s ataxia. This study will involve five visits to a research clinic over a 12-month period. Sites are open at Long Beach CNS, UCLA, University of Iowa, and University of South Florida. Click   here for the eligibility criteria and study schedule.

FARA's Director of Patient Engagement, Susan Walther recently conducted a Q&A with Retrotope's Vice President of Medical Affairs, Dr. Mark Midei, about their study drug. You can access that interview by clicking the image below.
Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular studies.
Cure FA Soirée
The Gehr family hosted their 3rd Annual Cure FA Soirée Virtually on Saturday, May 16th, 2020. Thanks to all who supported the Cure FA Soirée by donating, sponsoring, watching, performing, speaking, and participating in the silent auction. The Soirée raised an incredible $85,000 for FARA! If you would like, it's not too late to donate. Click here to make a donation.

If you missed it or would like to watch it again, here are the links to the full recording and the individual acts and speakers .
rideATAXIA NorCal Virtual Challenge
The rideATAXIA NorCal team is looking strong with $67,000 in fundraising and over 21,000 miles virtually traveld as a team in May! That's more than 7 cross country trips! It is all culminating on virtual ride day, this Saturday, where will celebrate the team accomplishments together on Zoom at Noon (Pacific). To register to join the Zoom gathering, click here. (We will also try to stream the gathering on rideATAXIA's Facebook page .) Go Team!
FA Research Flash Talk Series
In honor of FA Awareness Month, FARA hosted an FA Research Flash Talk series- featuring Young Investigators from FARA funded laboratories around the world. This five-part series covers key aspects of FARA funded research from gene and protein function to clinical outcomes and insights. Each session includes Flash Talks from three to four Young Investigators (ie, postdocs and graduate students).

Videos from our previous sessions are posted below. If you'd like to attend the last sessi on, click the Register button to sign up to receive meeting coordinates.

Click one of the topics below to watch the session:

  • Clinical Outcomes & Insights - May 28 at 7pm (EST). Click here to register.
FARA Engages in ARM Hill Day to Support
& Advance Our Mission
On May 21, FARA joined the Alliance for Regenerative Medicine (ARM) in virtual meetings with Members of Congress to discuss the need to remove the legislative hurdles that are blocking the adoption of value-based payment models in the Medicare and Medicaid programs.  
ARM is an international community of stakeholders dedicated to realizing the promise of regenerative medicine for patients around the world. Regenerative medicine aims to alter the current practice of medicine by treating the root cause of disease and disorders through gene and cell therapies. A challenge of delivering these treatments is devising a fair and affordable payment model which will enable access for all patients. Many value-based payment models that have been proposed run into roadblocks with current regulations. FARA’s support of efforts to educate and raise awareness of issues like this will help pave the road to access for future treatments.

Just as ARM’s Hill Day was virtual, many organizations are adapting their events to continue to serve the rare disease community during COVID-19. There are many wonderful opportunities for you to learn, connect or even advocate from the comforts of home! Here are some upcoming programs:

Global Genes and the Everylife Foundation is sponsoring Rare on the Road . This is a two-part virtual experience that will empower attendees to build their skill set, enhance their knowledge, engage in advocacy, and network with their local rare disease community.
  • Part 1: Interactive Webinar will be held on Tuesday, June 23rd, 11 am - 1:30 pm ET.
  • Part II: Rare Chats will be on Wednesday, June 24, 11 am - 12 pm ET.

There is an optional $25 registration fee. Deadline to register is June 15. BONUS: if you register by June 1, you will receive a RARE care package.

NORD is sponsoring 2020 Living Rare, Living Stronger, NORD Patient and Family Virtual Forum on July 18-19. This event brings rare patients and families together with health care professionals to network, educate, inspire and have fun. Learn from the experts, hear inspiring stories, sit in a wellness workshop and meet others affected by rare disease just like you. Patient pricing $39.

RDLA is sponsoring Rare Across America from Aug 3-Sep 7. Meet your Members of Congress and/or the Member’s staff while they are in district on recess. Last year, 600 rare disease advocates held 303 meetings in 49 states and DC resulting in increased Congressional support in many areas. Join the effort this summer! Virtual meetings may be possible and if that is your preference, make sure to note it in your registration. No prior experience is necessary, RDLA will prepare you for the meeting, provide legislative resource material, and host pre-meeting training webinars. There is no charge for this program.
Sign Up for the Patient Registry Today!
The goals of the FA Global Patient Registry (FAGPR) are to collect information on all FA patients in one registry, to develop the registry into a powerful resource for research, and to engage the FA community in studies aimed at advancing our knowledge of FA and the treatments being developed. This registry is only for people diagnosed with Friedreich's ataxia. Parents of minors diagnosed with FA can register an account on behalf of their children. Be sure you are included in the new registry. Click the button below to enroll and to engage with resources related to living with FA.