Your Community News
Dear friends,

As you know, Fall is the season for many FARA fundraising events, a scientific conference, and our largest educational FA Patient Symposium. We've been working on adapting those events to a virtual format, and we are excited to deliver all of their energy and information straight to your home. You will find the details for these events throughout this newsletter.

We also recently launched a new program called The Pathway - a monthly recurring gift program. In honor of the 1 in 50,000 people living with FA, we set a goal to enroll 50 new contributors in The Pathway over the next couple of weeks. A monthly donation of $10 or more is a budget friendly way to support ongoing research.

In the first week, 20 new participants enrolled in the program, and I am grateful for the opportunity to be one of them. I believe in the power of research to transform lives. FARA funds research along the entire continuum- from the lab bench to the clinical bedside. Sustaining contributions received through The Pathway allow FARA to ensure that researchers are exploring all molecular, systemic and clinical pathways that will lead to treatments and a cure with urgency.

As always, thank you for your support.


Jen Farmer,
Chief Executive Officer
Open & Enrolling Studies
Retrotope RT001-006 Phase 2/3 trial for protection against oxidative damage in the central nervous system
Retrotope is seeking 30 more 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 is enrolling participants age 12-50 and will involve five visits to a research clinic over a 12-month period. Sites are open at Long Beach CNS, UCLA, University of Iowa, University of South Florida, and University of Florida - Gainesville. For eligibility screening and additional study information, please contact the study coordinator, Sarah Endemann, at

FARA's Director of Patient Engagement, Susan Walther, conducted a Q&A with Retrotope's Vice President of Medical Affairs, Dr. Mark Midei, about their study drug. You can access the interview by clicking HERE.
Heart and Eye Study at Weill Cornell Medical College
This study is currently recruiting 20 participants ages 12-17 diagnosed with FA along with their unaffected siblings in the same age range. This study is designed to characterize the cardiac manifestations of FA using exercise, MRI, ECHO and blood tests. Additionally, this study will include corneal confocal microscopy (CCM), a non-invasive eye evaluation. Study participants and their siblings will have 2 visits in 1 year to Weill Cornell Medicine in New York City; rapid COVID-19 testing will be performed at the hospital prior to study visit. Financial support is available for travel and hotel. For eligibility screening and additional study information, please contact the study coordinator through email at
Phase I study of CTI-1601 - Larimar Therapeutics and Clinilabs Drug Development Corporation
Larimar and Clinilabs are seeking participants for several cohorts for their study of frataxin protein replacement. The next two cohorts will enroll in September and October. Eligible participants must be age 18 years or older with genetically confirmed Friedreich's ataxia and be able to go the distance of 25 feet with or without an assistive device or by pushing oneself using a manual wheelchair. This study includes an in-person stay of up to 22 days at a clinical research unit in Eatontown, NJ. For eligibility screening and additional study information, please contact the study coordinator, Rupal Patel, at or (212) 981-2715.
IDEA Study
The IDEA research study is testing 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, Los Angeles or Portland (OR). 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. 
New enrollments are on hold due to COVID-19, but phone screening can still be initiated now to prepare for the first clinic visit.
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. 
Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular study.
Newly Awarded FARA Grants
AAV-mediated therapy for visual impairment associated with Friedreich's ataxia
General Research Grant: Shannon Boye, PhD - University of Florida

Progress is being made to develop a whole body (systemic) gene therapy to treat FA. While systemically delivered Adeno Associated Virus (AAV) can efficiently target the muscle and heart, and injections into the spinal cord can efficiently reach the brain, neither are likely to mediate sufficient levels of therapeutic transgene in the retina to prevent vision loss. A more directed approach is needed to treat the ocular phenotype of FA. The purpose of this study is twofold; 1) to characterize the natural history of a retina specific Fxn knock out mouse model, and 2) to develop a retina-targeted, AAV-based therapy for preserving vision in FA patients. To learn more click HERE and scroll to "Gene and Stem Cell Therapy."
Save the Dates - United Against Ataxia Hill Day
The National Ataxia Foundation (NAF) and FARA will be holding their second Hill day on Thursday, September 10, 2020, to raise awareness about ataxia and support legislation that directly impacts our communities. In addition to the scheduled meetings NAF and FARA will have with US Senators, we are asking members of both communities to flood the hill with correspondences (calls, emails, social media posts) that day! Here is how you can take part!

Save the date to join an advocacy webinar on Wednesday, September 2 at 7 pm EST. At that time, members from NAF and FARA will review our priorities and review how you can make your voices heard. Click HERE for a quick highlight of the issues we are currently following or to register for the September 2 webinar.
rideATAXIA Global Challenge
This Fall rideATAXIA is moving from our neighborhood to yours with the first ever virtual rideATAXIA Global Challenge, and we want YOU to join us. Our goal is to log enough miles as a team to circumnavigate the globe, while raising funds and awareness for FA research, and we need your help!

Registration for the rideATAXIA Global Challenge is free and open now. We will officially kick off the month-long effort on International Ataxia Awareness Day- September 25, 2020. You can cycle, run, walk, wheel, stretch, do yoga, meditate, read- as long as your body, mind or spirit is active- you're in. We hope that you will find strength in rallying, as a geographically distant but still united team, to log miles, and dollars for research.
Friedreich's Ataxia Patient Symposium
Virtual Series
The Friedreich's Ataxia Research Alliance (FARA) and the Friedreich’s Ataxia Program at Children’s Hospital of Philadelphia are pleased to present this year's symposium as a virtual series of talks throughout October providing patients and families with up-to-date information about therapeutic approaches and current research being conducted in the field of Friedreich’s ataxia There is no fee to register. We hope you will join us for some or all of the sessions.

FARA Energy Ball- November 7
The 12th FARA Energy Ball will take place as a virtual event on Saturday, November 7, 2020. The program will be live streamed, and we hope you will tune in from home, perhaps even with a small viewing party of your own.

For more information, click HERE.
Now is the Time to Act
From our advocacy partner Research!America
Senate Republicans have a new legislative proposal for COVID-19 emergency supplemental spending which includes provisions such as extended unemployment benefits and emergency funding for USPS, but no supplemental
funding to restore COVID-19-eroded research dollars.

Speaking up now is essential if there is to be an emergency spending bill that fully fights back COVID-19's attack on the nations economy and health security. Reach out to your members of Congress to encourage them to include at least $26 billion in funding to restart lifesaving research supported by our health and science agencies.

Here are two ways you can act right away:

#RAREis Scholarship Fund
The EveryLife Foundation for Rare Diseases established the #RAREis Scholarship Fund – to enrich the lives of adults living with rare diseases by providing support for their educational pursuits. Thanks to the support of Horizon Therapeutics plc, one-time awards of $5,000 each will be granted to up to 32 recipients for the Spring 2021 semester.

Who can apply? Anyone over the age of 17 who are residents of the United States and have been diagnosed by a physician as having any form of rare disease regardless of treatment status. (Undiagnosed applicants may apply with a completed Diagnosis Verification Form by a physician).

The scholarship may be applied to a course of any subject matter; including vocational-technical/trade school, undergraduate, graduate study and single classes verified under an accredited program. The deadline to apply is August 28th at 3:00pm CST.
2020 RARE Artist Contest
The 2020 Rare Artist contest is now open for submissions! The Rare Artist Contest celebrates the talents of the rare disease community and spreads awareness with each piece and its artist. Anyone who is connected to rare is encouraged to enter, they accept entries from ages 4+.

To participate in the Public Vote, please register via Facebook. For more details on contest information or guidelines please visit

If you do not have a Facebook page, or are not interested in the Public Vote, please send your art submissions directly to