Dear friends,
We are gearing up for a busy fall and hope that you will find one or more opportunities to participate. In this newsletter, there is information about United Against Ataxia Hill Day, educational symposia and webinars, and many research studies in need of participants.
FARA and the Friedreich’s Ataxia Center of Excellence at Children’s Hospital of Philadelphia (CHOP) will host an in-person and virtual symposium September 24th and 25th. This symposium will includes talks and panel presentations on topics such as FA Clinical Management, Clinical Stage Programs in FA, and Gene Therapy and Gene Editing.
The symposium will be held at the Sheraton Valley Forge in King of Prussia, PA. For those attending in person, the symposium will begin with a welcome reception Saturday, September 24th from 5:00 pm - 6:30 pm (ET). The virtual and in-person general session and presentations will follow on Sunday, September 25th from 8:00 am - 4:30 pm (ET).
Thanks to the generosity of our sponsors, there is no fee to register for the FA Symposium. We hope to see you online or in person. Register here.
Sincerely,
Jen
Jen Farmer,
FARA Chief Executive Officer
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- Larimar plans to submit a complete response to CTI-1601’s clinical hold in the third quarter of 2022
- In conjunction with the complete response, Larimar is proposing a Phase 2, four-week dose exploration study in Friedreich’s ataxia (FA) patients as CTI-1601’s next clinical trial
Click here to read the full press release. |
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Reata Pharmaceuticals recently completed a mid-cycle communication meeting with the Food and Drug Administration (FDA) where feedback arising from the current review of the New Drug Application (NDA) for Omaveloxolone for the treatment of Friedreich’s ataxia was provided to Reata. At this meeting, the FDA agreed that Reata could submit updated supportive data to the existing NDA. With this data now submitted, the FDA has advised that they will require an extension to appropriately consider the updated application. The review is now due to conclude on February 28, 2023.
Click here to register for an informational webinar on these new developments with FARA CEO, Jen Farmer and study Principal Investigator, Dr. David Lynch on Tuesday, August 30th at 7pm (ET). Click the titles above to read the full press releases.
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FARA announced that data from a FARA-sponsored natural history study – the FA Clinical Outcomes Measures Study (FACOMS) -- has helped provide evidence and understanding of the treatment effect of Omaveloxolone, a potential treatment currently under review for approval by the Food and Drug Administration (FDA).
Jennifer Farmer, Chief Executive Officer of FARA, states, “This publication demonstrates the power of the natural history study in providing critical data to support clinical trials and shaving time off drug development. FARA is proud to have invested in and fostered this research. We are hopeful that this study will provide confirmatory evidence of the treatment effect of Omav observed in the MOXIe Part 2 study and can support an FDA decision to approve Omav for FA. In a progressive neuromuscular disease, we do not have the luxury of time and must operate with efficiency and urgency.”
Click here to read the full press release from FARA or the publication in medRXiv.
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The FARA Grant Program is excited to announce that the 2022 Award for Innovative Mindset has been granted to Manuela Corti, PhD and Tanja Taivassalo, PhD at the University of Florida for functional electrical stimulations (FES) cycling training to improve motor and cardiac functions in patients detection and enhancement of tissue NAD+ levels in Friedreich Ataxia. Learn more about this award and the grant program here.
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United Against Ataxia Hill Day
September 21, 2022
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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.
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Sign up for Hill Day HERE!- Registration will close on August 31, 2022
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Email brigid.brennan@curefa.org to obtain the recording of the one hour required training session
Important! Your Congressional meetings with not be scheduled until you complete the training.
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rideATAXIA Philly & rideATAXIA Dallas |
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Join us for rideATAXIA Philly on September 24th and rideATAXIA Dallas on November 5th! Registration for both of these events is open now. Come for a day filled with cycling, food, and community. Both events include multiple cycling routes, a walk option, and lunch generously catered by Outback Steakhouse.
- Register for rideATAXIA Philly here
- Register for rideATAXIA Dallas here
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Join us for the FARA Energy Ball on Saturday, October 15th!
*NEW LOCATION*
JW Marriott
510 Water St.
Tampa, FL 33602
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New Clinical Studies/Trials |
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Two new clinical studies/trials are open for enrollment.
1. Measure patient-relevant changes in disease burden over time FA-HI (Friedreich Ataxia-Health Index)
2. Measure caregiver-reported changes in disease burden over time FACR-HI (Friedreich Ataxia Caregiver Reported-Health Index).
The study will require completion of surveys (lasting 15-30 minutes) either by an FA patient or an FA caregiver at four timepoints- baseline, 6 months, 12 months, and 18 months.
Click the titles for additional information about each study.
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There are several clinical studies and trials open and enrolling participants. Click the titles below to learn more about the research studies, participating sites, and contact information for study coordinators. 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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