Dear friends,
In October, I usually enlist my local friends and family to assist the staff at rideATAXIA Philly near FARA'S home office. Th is year, rideATAXIA is moving from our neighborhood to yours with the first ever virtual rideATAXIA Global Challenge, and we want EVERYONE to join us. Our goal is to log enough miles to circumnavigate the globe in 30 days, while raising funds and awareness for FA research. As our participants log miles, we will travel virtually around the globe, via Google Earth, to "visit" different FA research and clinical locations and meet the people behind the research.
I am excited for the opportunity to bring together FA academic researchers, advocacy organizations, clinicians, industry partners, and the patient community. When we all work together as one team, we advance FA research with the sense of urgency needed to find treatments and a cure for FA.
I set an activity goal of to contribute 60 running miles to help get our team around the Globe this month. We launched the challenge on September 25th, but there is still time to join by clicking the button below.
As always, thank you for your support.
Sincerely,
Jen Farmer,
Chief Executive Officer
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A Few More Volunteers Needed to Complete Enrollment in October - Retrotope RT001-006 Phase 2/3 trial for protection against oxidative damage in the central nervous system
Retrotope is seeking 20 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, CA 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 [email protected].
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.
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Recruitment for final cohorts - Phase I study of CTI-1601 - Larimar Therapeutics and Clinilabs Drug Development Corporation
Larimar and Clinilabs are seeking participants for the final cohorts for their study of frataxin protein replacement. The next two cohorts will enroll in October and January. 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 [email protected] or (212) 981-2715.
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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 [email protected]
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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 ([email protected]) 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.
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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.
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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. |
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Newly Awarded FARA Grants |
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Awards for Innovative Mindset
FARA, the CureFA Foundation, and fara Australia are proud to announce the recipients of the Award for Innovative Mindset (AIM). AIM was designed to promote the exploration of high-risk, high-gain, and potentially groundbreaking concepts in FA research. The request for proposals was initiated in May, during Friedreich's Ataxia Awareness Month, and the research awards are supported in part by funds raised in the Lend Us Some Muscle campaign. The intent of the award was to inspire creativity, thoughtful reflection, innovation and to advance FA research, during a time when many investigators were away from their labs and daily routines due to the shutdown. Seventeen applications were received and four were awarded funding. A description of each project is available HERE.
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Elucidating the link between genome topology and repeat instability in Friedreich’s Ataxia
General Research Grant: Jennifer Phillips-Cremins, PhD – University of Pennsylvania
This group has uncovered a striking new link between 3D genome structure and trinucleotide repeat (TNR) expansion disorders, like Friedreich’s ataxia (FRDA). They have discovered that nearly all genes that cause TNR disorders are folded into the same unique 3D structure. This group proposes to create maps of 3D genome structure in cells from FRDA patients and quantify the FRATAXIN GAA repeat tract length. Knowledge gained by this work will empower the long-term goal to engineer the 3D genome to reverse gene expression defects in human disease. To learn more click HERE and scroll to "Mechanism or Pathway of Disease ".
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FA Biomarker and Clinical Endpoint Meeting |
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FARA hosted a virtual scientific meeting on September 24th and 25th to review the state of biomarker and novel endpoint development for FA. FARA’s goal is to have a toolbox of biomarkers and outcome assessments that can be used in various settings and for diverse treatment approaches.
The meeting hosted over 285 attendees from the scientific community, many from our academic, clinical and industry partners. Attendees had two days of thought-provoking presentations and heard recent advancements and exciting new avenues of clinical outcome measure and biomarker development in FA. A special thank you to our Scientific Steering Committee, Session Chairs, Speakers and Sponsors for making this a successful virtual meeting.
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United Against Ataxia Hill Day - Thank You! |
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#AtaxiaHillDay was a huge success! Thank you to all of the supporters who joined the advocacy webinar and then helped “flood the Hill” with calls, emails and posts to social media! Twenty Senate meetings were held with 47 advocates calling in from all over the country. Meetings included ataxia patients, physician/researchers, industry partners and members of both NAF and FARA. Eight letters supporting the National Ataxia Awareness Day resolution were also submitted by key stakeholders at institutions treating and researching ataxia.
Advocates found every Senate meeting to include staffers that were interested and engaged in learning about ataxia. Robust conversations were held throughout the day on funding for the NIH/FDA, coverage for telemedicine, the Creating Hope Reauthorization Act and the Rare Disease Congressional Caucus. The pinnacle moment occurred, however, when Republican Senator Cindy Hyde-Smith (MS) agreed to co-sponsor the National Ataxia Awareness Day Resolution with Democratic Senator Elizabeth Warren (MA)! We anticipate unanimous passage of this resolution within days for Friday, September 25 to officially become National Ataxia Awareness Day!
Let’s continue the momentum of our Hill Day! You now have a great reason to reach out to your Senators, and Senators Warren & Hyde-Smith, and thank them for their support!
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Friedreich's Ataxia Patient Symposium
Virtual Series
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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. The first session is October 1st at 7:00pm EST.
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Virtual FARA Energy Ball- November 7 |
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The 12th FARA Energy Ball will take place as a virtual event on Saturday, November 7, 2020 at 7pm. The hour-long program will be live streamed and include a silent and live auction. We hope you will tune in from home, perhaps even with a small viewing party of your own. For more information, click HERE.
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Non-partisan Election Resource for the Rare Disease Community |
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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.
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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.
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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 disease is encouraged to enter. Submissions accepted from age 4+.
If you do not have a Facebook page, or are not interested in the Public Vote, please send your art submissions directly to [email protected].
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