Dear Friends,
Every year in May, our community comes together in action for FA Awareness Month. This month is dedicated to hosting educational events and engaging in advocacy—efforts aimed at sharing information about Friedreich's ataxia (FA) and driving progress toward treatments.
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So many of you have taken the initiative to share information about FA on social media and engage with FARA. By sharing informative posts, personal stories, research updates, and upcoming events through online platforms, we can connect individuals, provide support, and foster a sense of unity––even when physical proximity is not possible.
FARA Ambassadors serve as a driving force behind FA Awareness, facilitating meaningful connections, providing valuable insights, and promoting collaboration among various stakeholders—individuals with FA, pharmaceutical companies, regulatory agencies, researchers, and healthcare providers. This month, ambassadors and community members are sharing their stories and experiences to raise awareness and promote patient engagement in research at events hosted by industry partners, including Larimar Therapeutics, Reata Pharmaceuticals, and Solid Biosciences.
Additionally, we are incredibly grateful for the individuals and families who have organized and actively participated in fundraisers, as their contributions play a vital role in supporting FA research and bringing us closer to finding effective treatments.
Let's make this FA Awareness Month a time to unite as a community, amplify our voices, and educate others about FA. Your action ignites lasting change and progress.
Sincerely,
Jen
Jen Farmer,
FARA Chief Executive Officer
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May is FA Awareness Month |
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FARA Flash Talks
In honor of FA Awareness Month this May, we proudly present the fourth annual FARA Flash Talks series featuring young investigators from FARA-funded laboratories worldwide. This four-part series covers critical aspects of FA research, from gene silencing and disease mechanisms to therapeutic approaches and clinical insights.
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These webinars serve as a catalyst for knowledge exchange, facilitating collaboration within the field and ensuring that valuable insights reach a wider audience.
Each session consists of four Flash Talks from postdocs and graduate students, moderated by FA community members. The talks are limited to five minutes and a single PowerPoint slide, making them suitable for a lay audience.
Register for the final 2023 Flash Talks session on May 25th here.
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Lend Us Some Muscle
Join us in the Lend Us Some Muscle (LUSM) Global Challenge this May to celebrate FA Awareness Day on May 20, 2023.
The goals of this initiative are to spread awareness, be active, and raise funds for FA research.
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We encourage you to show your support for people living with FA by staying active this May. Whether it's bicep curls, running, walking the dog, trying out yoga, or hitting the gym, let's do it for a great cause!
Share your accomplishments with the hashtags: #LendUsSomeMuscle #GetActiveInMayForFA #FlexForFA #FAAwarenessDay
Please consider making a $15 donation to fund research that supports the over 15,000 people affected by FA globally. Click here to donate.
Thank you to all who have already flexed their muscles and shared how they are staying active in May for FA on social media. We encourage you to keep sharing your active endeavors to spread awareness about FA.
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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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Walnut Creek, CA
Sun. May 21, 2023
1:30 pm - 6:00 pm PT
Embassy Suites
Walnut Creek
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Edmond, OK
Sat. June 10, 2023
10:00 am - 2:00 pm CT
Hilton Garden Inn Edmond*
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* Virtual option available for the Edmond, OK Research Reception. |
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Save the Date: FA Symposium – October 9, 2023 |
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FARA and the Friedreich’s Ataxia Center of Excellence at Children’s Hospital of Philadelphia (CHOP) will host this year's Friedreich’s Ataxia Symposium on Monday, October 9, 2023.
This symposium will begin with a welcome reception on the evening of Sunday, October 8. The general session and presentations will be Monday, October 9 from 8:00 am – 4:30 pm.
Save the date! Registration, agenda, and hotel block to follow.
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Additional Upcoming Events |
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For all upcoming events, please visit curefa.org/events. Special thanks to all of our volunteer event organizers and supporters.
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May 2023 | Virtual
FA Awareness Day
Sat. May 20 | Global
Sun. May 21 | Walnut Creek, CA
Thur. May 25 | Virtual
Sat. June 10 | Edmond, OK
Sat. June 10 | Edmond, OK
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Save the Date: FARA Energy Ball – October 14, 2023 |
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We hope to see you at the 2023 FARA Energy Ball! This year, we will return to the Tampa Marriott Water Street on October 14 for a high-energy evening of camaraderie and fundraising to support Friedreich's ataxia research. (Please also plan to join us for a Research Reception brunch, presented by FARA and the University of Florida, earlier that day.)
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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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Join us for rideATAXIA Chicago on Sunday, July 9th at Channahon Central Park.
Enjoy a 27-mile, 12-mile, or 4-mile ride or 1-mile walk with lunch to follow from Outback Steakhouse.
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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.
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Research Pipeline: Get Involved and Stay Informed |
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On May 10, 2023, Reata Pharmaceuticals provided an update on their development programs including SKYCLARYS (omaveloxolone) during their Q1 investor conference call. Updates on SKYCLARYS included:
- Reata has completed the final stages of SKYCLARYS drug product manufacturing and packaging. They have submitted supplemental information to the FDA on the previously reported manufacturing impurity which FDA is currently reviewing. Provided there are no major review issues, Reata believes SKYCLARYS will be available through the specialty pharmacy no later than mid-August 2023.
- The Marketing Authorization Application for omaveloxolone in Europe is under review. Reata anticipates being able to respond to the Day 120 List of Questions from EMA by Q3.
- Reata is evaluating strategies to support global label expansion to pediatric patients and is working with EMA and FDA to that end.
While we, at FARA, understand the disappointment in the news of a delay in drug availability, we appreciate the mechanisms in place at FDA to ensure safety before commercial supply is released to patients.
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On May 15, 2023, Larimar Therapeutics reported preliminary top-line data from their phase 2 trial’s 25 mg cohort showing increases in frataxin levels in patients with Friedreich’s ataxia. The report included the following:
- Safety data indicate that repeated subcutaneous injections of 25 mg CTI-1601 were generally well tolerated when administered daily for 14 days and then every other day thereafter until day 28.
- Daily subcutaneous injections of 25 mg CTI-1601 for 14 days led to increases in frataxin levels from baseline compared to placebo in all evaluated tissues (skin and buccal cells).
- Median placebo-adjusted increases from baseline of 3.5 pg/µg and 0.9 pg/µg in frataxin levels were observed in skin and buccal cells, respectively, with 14 days of daily dosing of CTI-1601.
- Larimar has submitted data to FDA and will meet with the Agency to discuss the next steps; an update is expected in Q3 2023.
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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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The goals of the FA Global Patient Registry (FAGPR) are to collect information on all FA patients in one registry, develop the registry into a powerful resource for research, and engage the FA community in studies aimed at advancing our knowledge of FA and the treatments being developed.
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