Your Community News
Dear friends,

I hope you and your family are finding ways to enjoy the summer while also staying safe. At FARA, we continue to advance a number of research projects, advocacy initiatives, and fundraising campaigns. I hope the information in this newsletter serves as one indicator of volume of activity still happening even in a new socially distanced reality. Opportunities to participate include clinical trials, the United Against Ataxia Hill Day, the virtual rideATAXIA Global Challenge, the FA Symposium series, the virtual FARA Energy Ball, and contacting your representatives to encourage funding for the National Institutes of Health. Keep engaging your community and keep us informed by posting on social media and tagging FARA using #cureFA.

Thank you for your continued support of FA research.

Sincerely,

Jen Farmer,
Chief Executive Officer
Open & Enrolling Studies
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 August and September. 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 rpatel@clinilabs.com or (212) 981-2715.
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 sarah@retrotope.com.

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 .
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.
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
Investigating sympathetic nervous dysregulation in the pathogenesis of cardiomyopathy in Friedreich’s ataxia
Postdoctoral Research Award: Jarmon Lees, PhD – St Vincent's Institute of Medical Research, Melbourne, Australia

Major hurdles in the development of effective therapies to combat Friedreich’s ataxia (FRDA)-associated heart disease include the difficulty of obtaining human heart tissue for study. To this end, disease modelling using human induced pluripotent stem cells (iPSCs) may provide a solution by delivering FRDA heart tissue in a dish. Clinical studies of FRDA have reported dysfunction of the heart’s autonomic nervous system, the electrical system that controls how fast or slow the heart beats. It is plausible that both the heart disease and arrhythmias, commonly manifested in FRDA patients, are linked to this dysfunction of the autonomic nervous system. In this proposal, Dr. Lees aims to investigate the causes of the heart disease in FRDA by generating cardiomyocytes and autonomic neurons from FRDA-iPSCs to study the development of FRDA-associated heart disease. Click HERE and scroll to "Cardiac Research" to read more.
 
Studying the role of white adipose tissue dysfunction in the development of metabolic complications in Friedreich’s ataxia 
General Research Grant: Katia Aquilano, PhD – University of Rome Tor Vergata, Italy

Patients with Friedreich's ataxia (FA) manifest an increased risk of developing type 2 diabetes (T2D). Since the expansion of white adipose tissue (WAT) plays a fundamental role in the onset of T2D, this group of investigators proposes to characterize white adipose tissue (WAT) in a mouse model of FA (KIKO), in order to test whether dysfunction of WAT contributes to the disease. A strict interaction exists between gut microbiota and WAT, as some members of the microbial community are able to release molecules (e.g. short chain fatty acids) that exert beneficial effects on WAT physiology. Therefore, these investigators will also evaluate gut microbiota composition in KIKO mice and FRDA patients. Click HERE and scroll to "Mechanism or Pathway of Disease" to research more.
The 2020 Ataxian Athlete Initiative Award
The 2020 Ataxian Athlete Initiative (AAI) is sponsored by a strong partnership between the Friedreich’s Ataxia Research Alliance (FARA), the Burrows Hill Foundation, Catrike, The Texas Irish Foundation, and UVA Sun Systems. The AAI is a unique program providing adaptive cycling equipment to people with ataxia who have demonstrated a strong desire to stay healthy and fit despite their progressive disabilities. 

“My disability may change how I stay active, but it can’t stop me from doing so. Having a trike means still enjoying physical activity. That is something that when I was diagnosed, I didn’t think I’d do again.” says 2020 AAI recipient Noah Griffith.

Click HERE to read the full press release including a full list of 2020 AAI award recipients.
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. 
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 will open next month. 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 clinical information on therapeutic approaches and current research being conducted in the field of Friedreich’s ataxia. Registration will open next month, and there is no fee to register. Session topics will include:

  • The COVID-19 Impact
  • FA: A Clinical Management Overview
  • Approaches to Therapy for FA and a Clinical Trials Pipeline Update
  • A Panel Discussion on Gene Therapy in FA
  • Exercise and FA
  • Keynote Talk: Jeremy Cowart, "I'm Possible"
Save the Date - FARA Energy Ball- November 7
For the health and safety of our donors, families and community, we have transitioned this year's in-person FARA Energy Ball to a virtual event. Please plan to tune in on November 7, 2020 for some high energy fun.
Third Party Data Breach
Recently, FARA learned of a data breach at Blackbaud Inc., a third-party data service provider we use for our fundraising efforts. FARA was one of many nonprofits and schools that was impacted by this incident.

Blackbaud informed us that they discovered and stopped a ransomware attack and, with the help of independent forensics experts and law enforcement, successfully prevented the cybercriminal from locking them out of the system and taking full control of their files. However, the attackers were able to remove a backup file that contained some personally identifying information about Blackbaud's nonprofit clients' donors and prospective donors, including those of FARA.  Please note that no credit card or bank account information was compromised in this breach.  Click  HERE  to read a full description of the incident on Blackbaud’s website. Click HERE to read the full notice from FARA.
Ask Congress to Support Rare Disease Patients in the Next Coronavirus Relief Package
Right now, policymakers in Washington are working on the next COVID-19 relief legislation. More than ever, Congress needs to take action to ensure that rare disease patients have access to safe and affordable health care, both for their routine treatment and any COVID-19 related care. 
 
 
Typically, rare disease patients receive care in hospitals, health centers, and doctor's offices. However, during the COVID-19 pandemic, it's increasingly important for rare disease patients to be able to receive their care through telemedicine to minimize their risk of contagion and serious illness. We ask that Congress make permanent changes to improve telehealth flexibility, to allow Medicare and Medicaid patients to continue to receive their treatment at home. 
 
In addition, Congress must increase funding for state Medicaid programs, so that they may continue to provide critical treatment and testing for the millions of patients who depend on Medicaid coverage.
Now is the Time to Act
From our advocacy partner Research!America
 
If your Senators and Representative in Congress don’t speak up now, funding to restore COVID-19-eroded research grants is in serious jeopardy.
 
It was reported this weekend that the Trump Administration opposes including funding for federal health agencies, including NIH, in the next supplemental spending bill. Negotiations on this legislation are reportedly near a close, and the bill could be introduced as early as this week.
 
At least $26 billion overall, and at least $10 billion for NIH, is needed to even begin to mitigate the damage COVID-19 has inflicted on research labs across our nation. The pandemic’s chilling effects have stalled medical and scientific progress that patients and our nation as a whole desperately need.
 
U.S. research capacity is at risk.
 
 
Note that the information gathered as part of this action alert is required by Congressional offices to verify that you are a constituent.
#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.