Dear friends,
Thank you to everyone who attended the FA Symposium in person or virtually earlier this month. If you were unable to tune in live, you can access the recording by clicking the image on the right. The playlist is organized in segments by presentation. We hope you find these sessions engaging and informative.
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Sincerely,
Jen
Jen Farmer,
Chief Executive Officer
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Design Therapeutics Webinar Tonight! |
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Design Therapeutics is developing gene targeted chimeras (GeneTACs™), which are designed to target the underlying cause of inherited nucleotide repeat expansion diseases to potentially modify the course of disease. The FA program is expected to begin a Phase 1 clinical trial in the first half of 2022 for people affected with Friedreich’s ataxia, age >18 years old.
Please join FARA and Design Therapeutics for an informational webinar tonight at 7:00 PM Eastern.
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Award for Innovative Mindset |
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In honor of FA Awareness Day in May, FARA (US) joined fara Australia's Lend Us Some Muscle campaign. Throughout the month, the community flexed its collective muscle not only in photos but also in fundraising. As a result, two research grants were funded under the Award for Innovative Mindset. Thanks to everyone who flexed, fundraised, and donated!
Nuclear frataxin and the regulation of macrophage activation
Marco Carpenter, PhD - Children’s Hospital of Philadelphia
Key evidence suggest frataxin could have a role beyond the mitochondrion and be important in the nucleus and involved in DNA repair. These observations encourage a new perspective on frataxin, such as a role in the regulation of DNA protection from oxidative damage and transcriptional regulation, and suggest that these functions could be important in the pathogenesis of FA. Dr. Carpenter proposes to optimize methodologies to profile and quantify frataxin interactions in the nucleus. This approach is the first to investigate nuclear frataxin-mediated gene regulation in response to increased oxidative stress. Findings from this proposal will inform the design of novel therapies targeting nuclear frataxin.
Co-sponsor: fara Australia
Dentato-Thalamo-Cortical tracts in Friedreich Ataxia: impact of its modulation on Friedreich Ataxia symptoms and brain functional architecture
Gilles Naeije, MD, PhD - Hôpital Erasme, Université libre de Bruxelles, Belgium
Cerebellar atrophy in FA also disrupts important connections between the cerebellum and the brain cortex and this degeneration is related to the onset and progression of motor and non-motor symptoms. Cerebellar transcranial direct current stimulation (ctDCS) is a non-invasive and clinically friendly technique that has shown efficacy in improving motor and cognitive performances in degenerative ataxia and is based on applying a weak electrical current to the scalp. The aim of this project is to assess the potential efficacy of ctDCS to alleviate FA symptoms and to understand the connections between the cerebellum and the brain cortex.
Co-sponsor: fara Australia
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Roadmap to Readiness- Gene Therapy
In preparation for potential clinical trials for gene therapy in 2022, FARA has developed educational modules to assist the FA community for this approach to treatment.
There are complex issues to consider that are different from the issues in traditional investigational drug trials. FARA invites you to engage in reviewing several education modules. Each module is self-paced and includes check-in exercises or worksheets to confirm your understanding and to allow you to keep this information for future reference.
We are excited to bring you this education and welcome your feedback! Information on clinical trials and gene therapy education can be accessed here. |
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rideATAXIA Dallas returns on November 6th! Join us in person at UNT Discovery Park in Denton, Texas.
There will be 6, 27, and 50-mile bike rides, a delicious post-ride meal provided by Outback Steakhouse, and time to enjoy the company of good friends.
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Upcoming Grassroots Events |
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*This study has new expanded inclusion criteria*
27 FA participants and 17 unaffected siblings are needed to fill this study by the end of 2021. The outcomes of this study are critical for allowing remote data collection during future clinical trials.
Eligibility criteria include:
· Early-onset participants - 12-30 years of age with FA-onset between age 5-25
· Late-onset participants - 26-60 years of age with FA-onset between age 25-40
· Participants must be able to walk 10-yards independently, without an assistive device.
· Participants can be involved in an investigational drug trial and still be eligible for the 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 site. Involvement in the study provides compensation for travel and an Apple watch.
Participants are needed at all clinic sites in Baltimore (Johns Hopkins), Boston (MGH), Chicago (University of Chicago), Los Angeles (UCLA) or Portland (Oregon Health & Science University).
For additional eligibility criteria, please contact study coordinator Hannah Casey (hannahcasey@uchicago.edu) or (773) 702-4610. Please specify your city of interest for enrollment - Baltimore, Boston, Chicago, Los Angeles or Portland (OR).
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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.
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Sites are open for enrollment at University of Minnesota (Minneapolis), University of Florida (Gainesville), Children’s Hospital of Philadelphia, Monash University (Victoria, Australia) and RWTH Aachen University (Aachen, Germany).
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. To contact a study coordinator, please click HERE.
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Enrollment is now closed for this study and capacity has been reached at each study site. Please email Susan Walther, Director of Patient Engagement at FARA, if you have any questions (susan.walther@curefa.org)
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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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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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The Pathway
You can help fund research progress all year by joining our monthly giving program, The Pathway. When you join The Pathway with a monthly donation of $10 or more, you are joining a community invested in setting a path to treatments and a cure for FA through research. Click the image to give today.
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