Your Community News
Dear friends,

It is with great excitement that I write to you about the upcoming FARA Energy Ball. While this event like many others is going to be different this year, it also opens up the opportunity for more people to participate in the Energy Ball than ever before.

On Saturday, November 7th, FARA will be hosting the Virtual 2020 FARA Energy Ball. The pre-show will begin at 6:30pm EST followed by the main event Broadcast from 7:00pm - 8:00pm EST. I hope you will join us for a #partyatyourhouse and consider participating in the festivities in one or more of the following ways.


  • Participate in the Silent Auction - Register now and start bidding on over 100 silent auction packages right from your smart phone (requires a US mobile number, we recommend registering on your computer and bidding on your mobile device). 

  • Spread the word! Share this information with anyone in your community that you think would like to participate.

With five days to go I can feel the momentum building for a great event! Your support will continue to move meaningful FA research forward at an urgent pace; bringing individuals with FA closer to treatments and a cure.

Sincerely,

Jen Farmer,
Chief Executive Officer
Open & Enrolling Studies
Exercise Study at CHOP and Penn
Researchers at the Children’s Hospital of Philadelphia and the Hospital of the University of Pennsylvania are recruiting participants (male and female, between ages 10-40, inclusive) for a study to test the effects of a dietary supplement combined with exercise on aerobic capacity. Aerobic capacity refers to the body’s ability to take in, transport, and use oxygen during exercise. Eligibility includes documented genetic diagnosis of FA, ability to complete exercise testing and not pregnant for women of child-bearing years.

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. You will be compensated for your time.

To learn more about the study and determine your eligibility, contact our study coordinator, Anna DeDio, by email dedioa@email.chop.edu or by phone [(267)-425-1998].
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 cora@med.cornell.edu
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 (hannahcasey@uchicago.edu) 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
Frataxin Deficiency as a Cause of Endothelial Senescence and Vasculature Remodeling in Friedreich's Ataxia
General Research Grant: Stephen Chan, MD, PhD - University of Pittsburgh School of Medicine

While most of the research in FA has focused on nerves and heart muscle, alterations in blood vessels of the heart may worsen the disease in FA. But, the role of FXN in these blood vessels has never been defined. Dr. Chan and his team propose that FXN deficiency in FA causes DNA damage in lung and heart blood vessel cells to promote heart disease. This group will study blood vessel cells originating from stem cells of patients with FA, coupled with analyses of heart tissue in FA patients and mouse models. To learn more click HERE and scroll to "Mechanism or Pathway of Disease".
Cardiac Benefit of Drugs that Stimulate Nrf2 and HCA2 pathways in Friedreich's ataxia.
General Research Grant: Elena Dedkova, PhD - University of California, Davis

Ixchel Pharma has developed a novel chemical entity, IMF, that increases frataxin, iron-sulfur and mitochondrial functions in a mouse model of FA. Furthermore, IMF dosing in these mice rescues their cardiac pathology and increases survival in the frataxin MCK-Cre mouse, which only lacks frataxin in the heart, and dies because of this loss. Because IMF could target similar pathways as RTA408/Omaveloxolone, this investigator proposes to compare efficacy of IMF and RTA408 in FA mouse models. To learn more click HERE and scroll to "Drug Discovery".
Development of Autologous Transplantation of Genetically Corrected Hematopoietic Stem Cells for Friedreich Ataxia
General Research Grant: Natalia Gomez-Ospina, MD, PhD - Stanford University

Previous studies have suggested that bone marrow transplantation can improve symptoms in a mouse model of FA. Dr. Gomez-Ospina proposes to investigate the application of blood stem cell transplantation to treat FA by: 1) Performing stem cell transplantation experiments into a new mouse model of FA and assessing the molecular and functional outcomes, 2) Using genetic engineering to investigate whether expression of the protein Frataxin is required in specific blood cell types for the therapeutic benefit of stem cell transplant, and 3) Establishing an in vitro model to assess if cellular bodies called mitochondria undergo inter-cellular transfer to confer function to neighboring cells. To learn more click HERE and scroll to "Gene and Stem Cell Therapy".

Co-Funded with FARA Ireland
Giving Tuesday - 2020
Mark your calendar, Giving Tuesday is December 1, 2020! FARA will be participating in this annual celebration of giving and we hope you will join us to learn more about how you can give the gift of research this year!

For more information follow-us on Facebook and Instagram - @curefa_org.
2020 Virtual FARA Energy Ball
Silent Auction is Open
The FARA Energy Ball's silent auction is open for bidding on the Qtego mobile bidding platform. You can now register and start bidding on over 100 auction packages right from YOUR SMART PHONE. Please share the link below with anyone interested in the auction and supporting Friedreich's ataxia research!

Mark your calendar to tune in for the Virtual FARA Energy Ball on November 7th with a pre-show starting at 6:30pm (EST) and the main broadcast at 7pm (EST). Bidding for the live auction packages will begin the day prior to the event and culminate during the live broadcast. The closing time for the silent auction will be Sunday, November 8th at 12pm (EST). You can watch the live stream right from your personal bidding link or on FARA's YouTube Channel.
rideATAXIA Global Challenge
The rideATAXIA Global Challenge had participation from over 600 registrants from 19 countries who formed 62 teams. Together, we traveled around the world nearly twice, and we exceeded our fundraising goal by raising over $458,000 for research! This experience has shown us all just how powerful we are when we act as a team toward a singular goal. Thank you to everyone that participated!
FA Patient Symposium Virtual Series
FARA and the Friedreich’s Ataxia Program at Children’s Hospital of Philadelphia hosted a FA Patient 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.

We had 400 attendees registered from over 20 different countries. The series was recorded and will be available soon on our website. For more information contact jamie.young@curefa.org
U.S. Rare Disease and COVID Vaccine Survey
Our partners at The EveryLife Foundation, in partnership with the Community Congress COVID-19 Response Working Group are conducting a survey to better understand the rare disease community’s perceptions and information needs about a vaccine for COVID-19 once one is approved.

Please consider taking 10-15 minutes to fill out this survey.
It will help ensure EveryLife and partners in the rare disease community can:

  • Represent your concerns related to a safe and effective vaccine in advocacy efforts
  • Advocate for the importance of transparency and information sharing about the benefits and risks associated with a vaccine
  • Understand how the rare disease community will approach decision making when a vaccine is available
  • Advocate for ongoing policy changes that will help keep the rare disease community connected even as a vaccine is rolled out and COVID-19 protections are rolled back.
Non-partisan Election Resource for the Rare Disease Community
As November approaches, the Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation, want to ensure the rare disease community’s voice is heard in local, state and federal elections around the country. That’s why the RDLA has compiled the RDLA Voting Resource Guide. This list of non-partisan resources is designed to assist advocates in learning about the voting process and how to educate candidates on the issues impacting the rare disease community. 
The EveryLife Foundation for Rare Diseases has announced a big change in programming for 2021. Rare Across America will now take place virtually from Feb 22 - Mar 5 and Rare Disease Week is moving to July 19-22 in Washington, DC.