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News & Press Releases

Here you will find the most recent organizational news from FARA, including information on events, and awareness and advocacy initiatives. To locate an article from a certain date, please use the archives on the right side of your screen.

Advocacy Newsletter - February 2023

Advocacy Newsletter - February 2023


Rare Disease Day will be recognized on February 28, 2023 and celebrations are currently underway. Below is an amazing list of events, throughout the world, that you can participate in to help raise awareness of rare disease in general, and FA specifically! Moreover, sharing your experience is the key to lawmakers understanding FA, and paves the road to legislative actions that can expedite treatments. Don’t let this Rare Disease Day pass without being part of the conversation!

The FA community has witnessed the impact of their advocacy in many ways, as detailed recently in FARA’s December Advocacy Newsletter, but none are as demonstrative as the work done regarding the Congressionally Directed Medical Research Program (CDMRP). The CDMRP is a great example of how the FA community created action through advocacy.

Each year, during the budget cycle, the US Congress selects conditions eligible for this grant program. The process to get on the list starts when a Congressional Member receives an “appropriation request” from a constituent. Basically, someone needs to ask for it! Each Member then reviews the appropriations requests they receive and decides which ones to forward on to the appropriation committee for further consideration. FA community stakeholders throughout the US, working in collaboration with FARA and the National Ataxia Foundation (NAF), not only made the request but also followed up through meetings, emails and calls to explain the relevance of FA to the goals of the CDMRP program as well as the potential benefits of investing in FA research.

The result: FA was added to the CDMRP in Fiscal Year (FY) 22, resulting in six FA researchers being recommended for funding totaling over $14 million! These grants are a direct result of the amazing advocacy done by this community!

Let’s keep the momentum going in 2023! There are many great Rare Disease Day events listed below. Pick one that works for you and start your year of FA advocacy!

Read the Full Advocacy Newsletter

Monthly Update - January 2023

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Dear friends,

In FARA's strategic plan, one of the core pillars is to share knowledge and know-how within the research and patient/caregiver communities. Some of the resources previously developed by FARA to help educate the FA community about clinical research include a series of self-paced Gene Therapy Learning Modules as well as a Clinical Trials Guide for Participants.

To add to this growing library of education materials, FARA recently created a Rare Disease Medication Frequently Asked Questions (FAQ) document. As programs advance through the Friedreich's Ataxia Treatment Pipeline, FARA has been researching how other rare disease patients access their approved medications. During this research, we learned some new vocabulary along with processes and programs. We outline some of our learnings in this FAQs document. We hope you find it useful. As we continue to identify education opportunities and resources, we are committed to sharing that information with the FA community.


Jennifer Farmer,
FARA Chief Executive Officer

Other topics in our January Monthly update:
  • Pharma News
  • Open & Enrolling Studies
  • Minutes of Science
  • Recently Funded FARA Grants
  • Advocacy in Action!
  • Save the Dates - rideATAXIA 2023

Read the Full Monthly Update

FA Monthly Research Update - Jan 2023

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This month's Research Update covers the following topics:

  • Upcoming FARA Research Grant Deadlines
  • 2022 FA Research Highlights
  • Pharma News
  • Recent FARA Funded FA Publications
  • Recent FA Publications
  • FARA Newly Awarded Research Grants
  • FARA Forum Webinars
  • Grant Opportunities
  • Meetings of Interest to the FA Community


Read the full Monthly Research Update

Informational Q&A session with Design Therapeutics on DT-216

Join us for an Informational Q&A session
with Design Therapeutics on their DT‑216 Program in FA

FARA and Design Therapeutics will host an informational Q&A session on the DT-216 Program in FA, on Wednesday, January 18, 2023 at 12pm (ET), for the FA patient community. The session will be a Q&A between FARA Ambassador & Board Director, Alex Fielding and Design Therapeutics President and Chief Executive Officer, Dr. Joao Siffert; and the Q&A will be moderated by FARA Director of Patient Engagement, Layne Rodden.

This session will provide information on the DT-216 program, including a review of the recently announced data from the single ascending dose portion of the Phase 1 trial as well as next steps for the Phase 1 trial and the program overall. To read the recent press release from Design Therapeutics, click HERE. Please submit your questions in advance with your registration for the event. Register HERE for the session. Submit your questions HERE. After registering, you will receive a confirmation email with the link to join us.

Register for the Session

Submit Questions

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.

Dr. Stepanova Presented with $20,000 Research Grant from Uplifting Athletes


Alyssa Zinar, Uplifting Athletes
(610) 506-2533

Felicia DeRosa, FARA
(484) 879 6160

Weill Cornell Medical College’s Dr. Anna Stepanova
Presented with $20,000 Research Grant from Uplifting Athletes

Dr. Stepanova is among 10 rare disease researchers to be awarded a grant at Uplifting Athletes’ Sixth Annual Young Investigator Draft presented by CSL Behring on Feb. 4 at Lincoln Financial Field

DOWNINGTOWN, PA (January 10, 2023)Uplifting Athletes, a nonprofit organization dedicated to serving the rare disease community, has announced that Dr. Anna Stepanova, Weill Cornell Medical College, will receive a $20,000 research grant as a member of Uplifting Athletes’ 2023 Young Investigator Draft Class.

Dr. Stepanova is among a group of 10 researchers who will each receive an unrestricted grant for medical research at Uplifting Athletes’ sixth annual Young Investigator Draft presented by CSL Behring on February 4, 2023 at Lincoln Financial Field in Philadelphia.


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