Dear Friends,
FARA believes education and community building are crucial to our mission. We are excited to announce a series of free, in-person educational events for individuals and families affected by FA. These FA Research Receptions and Symposia will provide attendees with the latest information on FA research and an opportunity to connect with others in the community.
We are looking forward to hosting these events in multiple locations across the United States. Information on the first three events coming up this spring and summer is below, and there will be a few additional events later this year in the fall that we will share in future updates. We are committed to sharing important research updates, providing education about FA, and gathering to meet and share FA experiences. We look forward to seeing you at one of these events and sharing our progress toward our goal of slowing, stopping, reversing, and, ultimately, curing FA.
Sincerely,
Jen
Jen Farmer,
FARA Chief Executive Officer
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Spring into Action: Join us at Upcoming Community Events |
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FARA is pleased to present our 2023 Research Reception series. These receptions are free, in-person educational events for the FA community. |
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Registration is now open for the following events: |
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Carlsbad, CA
Sat. April 22, 2023
1:30pm - 5:30pm PT
The Westin Carlsbad Resort and Spa
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Walnut Creek, CA
Sun. May 21, 2023
2:00pm - 6:00pm PT
Embassy Suites
Walnut Creek
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Edmond, OK
Sat. June 10, 2023
10:00am - 2:00pm CT
Hilton Garden Inn Edmond*
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* Virtual option available for the Edmond, OK Research Reception.
Details to follow for Fall 2023 education events including the Tampa, FL Research Reception and the FA Symposium (King of Prussia, PA).
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Sat. April 22 | Tampa, FL
Sat. April 29 | Huntsville, TX
Sat. May 6 | Smithfield, RI
Sun. May 7 | New York, NY
Sat. May 13 | Seaside Park, NJ
FA Awareness Day
Sat. May 20 | Global
For all upcoming events, please visit curefa.org/events. Special thanks to all of our volunteer event organizers and supporters.
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Pedal for a Purpose: rideATAXIA News & Updates |
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Can’t make it to one of the main rideATAXIA locations? Join rideATAXIA Hometown! rideATAXIA Hometown offers teammates the opportunity to ride individually or host local events for their community as part of the rideATAXIA Program Series. There are several Hometown options to choose from based on your event and cycling experience. |
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Apply to the Ataxian Athlete Initiative |
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The Ataxian Athlete Initiative (AAI) provides adaptive cycling equipment to people with ataxia who have demonstrated the desire to stay active and healthy despite their disabilities.
2023 AAI applications will be available online and accepted from February 1 through May 1. Awards will be announced by June 1, 2023.
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Be Your Own Hero: Take Control of Your Care |
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Don't Lose Your Medicaid Coverage |
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Medicaid is a lifeline for countless Americans, and many people living with neuromuscular disease rely on Medicaid for access to essential medical care. However, as the federal Public Health Emergency (PHE), declared during the COVID-19 pandemic, winds down, those on Medicaid might inadvertently lose their coverage. |
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Clinical Management Guidelines |
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We are excited to share the recently updated Clinical Management Guidelines for Friedreich Ataxia (FRDA).
The Guidelines include chapters on emergency care, pain management, family planning, and other relevant health information pertaining to living with FA.
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Please view the website for the full disclaimer and intended use. |
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Within the REACH Patient Center, you can find more information about the Start Form, speak with Care Navigators, learn about the specialty pharmacy's role in medication delivery, and explore affordability options.
Additionally, REACH has a Provider Center where healthcare providers can find more information about completing and submitting the Start Form to enroll patients for the REACH program.
On Thursday, April 20, 2023, Reata is hosting a pre-recorded webcast featuring an interview with FA community member, Matt LaFleur about his journey with FA. You can register for this webcast here.
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Research Pipeline: Get Involved and Stay Informed |
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Open and Enrolling Studies |
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.
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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 studies. |
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Can we find ways to substitute frataxin? |
The FARA Grant Program is pleased to award a grant to Benoit D’Autreaux, Ph.D. at CNRS-CEA-Université Paris-Saclay. With funding from a previous FARA grant, Dr. D’Autreaux developed a system to screen a library of hundreds of thousands of existing molecules to find substitutes of frataxin function. This follow-up grant will allow him to continue his work. He and his team will use computer algorithms to identify potential target molecules. Then, he will test these targets in cells (in vitro). If they look promising in the test tube, he will test them in fruit flies (in vivo).
This work has the potential to teach us more about frataxin, which is important in the identification of new therapeutic opportunities.
FARA is co-funding this grant in partnership with fara Australia.
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How does FA impact the brain's immune system? |
The FARA Grant Program is delighted to award a grant to Massimo Pandolfo, MD and Stefano Stifani, Ph.D. at McGill University in Quebec, Canada, to investigate how Friedreich’s ataxia (FA) affects the brain’s immune system.
Did you know that the brain and central nervous system (CNS) contain specialized immune cells called microglia? Microglia act as the first line of defense against harmful substances and pathogens that might enter the brain. These cells serve as a cleanup crew and are crucial to protecting and maintaining the health and function of the CNS.
Microglia serve a protective function for the brain, but they also promote inflammation because they are immune cells. The research team will explore if microglia are sensitive to the decrease in frataxin exhibited in FA. It is essential to understand if FA causes these immune cells to malfunction and how this possible malfunction might cause excess inflammation and affect neurons (brain cells). To conduct these experiments, the researchers will use stem cells derived from skin or blood cells to differentiate microglia and test their effect on neurons in vitro (in cells).
This work has the potential to teach us more about the types of cells that contribute to the pathology of FA beyond the tissues of the brain, spinal cord, and heart. Understanding the role of microglia and inflammation in FA is vital to identifying potential therapeutic targets and developing effective treatments.
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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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