Your Community News
Dear friends,

The 'Virtual Research' section of the FA App has gone live with the first academic research trial that you can enroll in and participate in directly via your phone. 

Log in to the FA App and click on 'Research' and 'Virtual' and see the new study which has been posted. If you have removed The FA App from your phone, you can reinstall from the App Store or Google Play and login. 

This first study required 15 FAers and 15 control subjects per age bracket. Thanks to all the FAers who have already enrolled and helped the study to reach its target for participants with FA in just a few weeks. The study is in need of more control subjects. We anticipate more virtual research opportunities in the future and new features based on community feedback.

Sincerely,
Jen

Jen Farmer,
FARA Chief Executive Officer
Recently Funded Grants
The FARA Grant Program is excited to announce that the 2022 Kyle Bryant Translational Research Award has been granted to Shana McCormack, MD, at the Children's Hospital of Philadelphia and Joseph Baur, PhD at the University of Pennsylvania for detection and enhancement of tissue NAD+ levels in Friedreich Ataxia. Learn more about this award and the grant program HERE.
FA Woodstock
Thank you to the Hook Family and their community of volunteers for hosting FA Woodstock at Flying H Ranch! FA families from near and far enjoyed fun-filled days of tie-dying, relaxing in the pool, spending time with friends and endless ice cream! In addition to all of the fun at FA Woodstock, attendees had the unique opportunity to participate in FA research.

Click HERE to read a great event recap and heartfelt tribute to FA Woodstock and the Hooks written by FA Community Member, Dillon Loomis-Head.
Minutes of Science
Ever wonder about the impact of your support on FA research? FARA, in collaboration with members of the FA community, created a series of short, animated videos to help explain how your research dollars are put to work. The latest episode in this series of videos can be found here. Our narrator, Heidi Bromley, talks about the clinical studies of hand and arm function in FA funded by your support. Look for a new episode every month.

Watch all of the Minutes of Science episodes on FARA's YouTube page.
NIH BRAIN Initiative Challenge
"Ethical Considerations of Brain Technologies"
What does the future look like? Jake Juip, a 16-year-old with Friedreich’s Ataxia, shared his perspectives about Ethical Considerations for Brain Technologies in his video submission to the NIH’s BRAIN Initiative Challenge. 

Jake’s video was selected as a Tier 1 Winner! In addition to receiving a federal prize, he also had the opportunity to participate in a lively discussion with Drs. John Ngai, Director of the NIH BRAIN Initiative, Walter Koroshetz, Director of the National Institute and Neurological Disorders and Stroke, Debara Tucci, Director of the National Institute on Deafness and Other Communication Disorders, and Richard Hodes, Director of the National Institute on Aging. 

During the discussion, Jake shared his journey with Friedreich’s Ataxia, including losing his ability to walk, and his key takeaways for neuroethical considerations. Jake spoke about the need for researchers to move boldly and swiftly to advance breakthroughs in medical research. He pledged to collaborate with urgency, transparency, and mutual respect to bring the patient experience to clinical research.

Congratulations on this well-deserved honor, Jake! Watch Jake's winning video HERE!
Thank you to the Gehr Family, the Soirée Team, the OK FA Community, and all of the supporters of the Cure FA Soirée! Over 200 guests (including members of 25 different FAmilies!) came together for a night of music and friendship - raising an astounding $230,000 for FA research! You can view the event's performances HERE.
Registration Open for
United Against Ataxia Hill Day
September 21, 2022
Your Congressional Members need to hear from you to fully understand what Ataxia is and what legislative action is needed! Join us in raising the awareness of Ataxia, needed research funding and ways to expedite treatments. The power to create change starts with you!
 
Please join us for the fourth United Against Ataxia Hill Day on September 21, 2022. All meetings will be virtual and run approximately 15-30 minutes in duration. You will be assigned 2-5 meetings between 9 am and 5 pm.

Registration requires two steps.
 
  • Sign up for Hill Day HERE!- Registration will close on August 31, 2022
 
  • Sign up for a one hour required training on August 10 at 7 pm ET HERE!

Important! Your Congressional meetings with not be scheduled until you complete the training.
The FARA Energy Ball
Join us for the FARA Energy Ball on Saturday, October 15th!

*NEW LOCATION*

JW Marriott
510 Water St.
Tampa, FL 33602


To view the full FARA Energy Ball invitation or to reserve a table, click HERE or email: ava.faraenergyball@curefa.org 
Branding Science Survey
Branding Science Group would like to explore the types of services and technologies that would be most helpful to communities that could benefit from different types of home technology. The purpose of their short online survey is to better understand how individuals with FA and others with mobility/dexterity issues use devices/technology at home to help complete daily tasks, as well as what unmet needs there are for technology in the home. If you would like to participate, please click HERE to get started. 

After the full results have been compiled, they will share a summary of the results. Should you agree to participate, and you match the survey requirements, the survey itself will take approximately 10-15 minutes to complete.
MPR News: Rare disease research at the
University of Minnesota holds global promise


Check out this great story about FARA Ambassador, Mekayla Holm and the TRACK-FA neuroimaging study on MPR News! You can find out more about the TRACK-FA study below. Special thanks to reporter and longtime FAmily friend, Sarah Gelbard, for this feature!
Open & Enrolling Studies
 
Please consider enrolling in this important study. Participants needed at all study sites.

TRACK-FA is a natural history study to investigate brain and spinal cord changes in individuals with Friedreich ataxia. If imaging differences in the central nervous system are correlated with progression of FA, these differences could be used in future clinical trials to monitor treatment outcome.

Eligibility criteria include:
  • Age >5 years with genetically confirmed diagnosis of FA.
  • GAA repeat expansion >55 in length on both alleles of the FXN gene or GAA repeat expansion on one allele and another mutation type on the other allele (e.g., point mutation, gene deletion).
  • Age of disease onset less than 25 years.
  • Disease duration less than 25 years.
  • Able to undergo MRI
  • Participants can be involved in an investigational drug trial and still be eligible for the TRACK-FA study.

There are 3 study visits, each approximately 12 months apart. At each study visit, participants will be asked to complete neurologic and functional testing, blood draw and brain and spinal cord MRI scan.
 
Compensation is provided for time and travel expenses. Watch an informational webinar here.

Sites are open for enrollment at University of Minnesota (Minneapolis), University of Florida (Gainesville), Children’s Hospital of Philadelphia (Pennsylvania), RWTH Aachen University (Aachen, Germany), University of Campinas (Brazil), and Monash University (Victoria, Australia). To contact a study coordinator, please click here. 

Researchers at Vanderbilt University Medical Center in Nashville, TN want to learn more about changes in glucose control and cardiac features in children with Friedreich's Ataxia. They are recruiting participants (ages 7-17 years) with FA for a study to determine if there is a relationship between blood sugar abnormalities (risk of diabetes) and cardiac changes.

If your child is selected to participate in this study, your child may complete blood testing, oral glucose tolerance testing (with a non-FDA approved stable isotope product), placement of a continuous glucose monitor, echocardiogram (ultrasound of the heart), and dual-energy X-Ray absorptiometry (DXA, x-ray scan) during a 1-2-day study visit. You and your child will be compensated for your time. Click here for more information about this study.

This is a first-in-human, Phase 1 study of DT-216 which means that trial will begin with single dose cohorts to assess safety. Enrollment will be in cohorts (typically 5-8 people). After each cohort is enrolled and tested, there will be a review of the safety, then typically several weeks later the next cohort will be enrolled to test the next dose. After the single dose study, there will be a multiple dose study, which is also a safety study. Multiple dose studies typically assess safety over a period of several weeks to a month. More information on the multiple dose study will be available once the single dose study is further along.

Inclusion criteria:
  • Individuals with FA ages 18 to 55 years old (inclusive) at screening
  • Genetically confirmed diagnosis of FA with homozygous GAA repeat expansions
  • Weight between 90 and 200lbs (approximately)
  • Ability to sit upright with thighs together and arms crossed without requiring support on more than two sides
  • Ability to perform basic daily care such as feeding yourself and basic personal hygiene with minimal assistance
  • Must have completed full COVID-19 vaccination at least 4 weeks before treatment

This study is being conducted at Clinilabs and will require about a week long stay at the study site. Compensation will be provided for qualified participants and caregivers. Individuals and caregivers who are not local to the research facility will receive no-cost transportation and accommodations. Click here for more information and to read the study flyer.

If you are selected to participate in this study, you would complete 2 in-person study visits, each lasting 2 days, over a 12-week study period at CHOP and Penn. During the study period, you may complete blood testing, exercise testing, administration of a dietary supplement, MRI scans, oral glucose tolerance testing (with a non-FDA approved stable isotope product) and optional muscle biopsies.
 
Participants will be compensated for time in the study, and participants will be provided with a recumbent bike, which they can keep after study completion.

To learn more about the study and determine your eligibility, contact the study coordinator, Anna DeDio, by email dedioa@email.chop.edu or by phone
(267) 425-1998.
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.
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.