Jumpin' for Jazz

03/02/2026

The FARA Grant Program is proud to award a Postdoctoral Research Award to Agostina Di Pizio, PhD, at CNR Institute of Neuroscience (IN-CNR) to study how frataxin loss affects sensory neurons and explore potential therapeutic strategies for FA.

Learn more about this grant and other FARA-funded research at https://ow.ly/sQ2p50YhGUh

03/02/2026

Let us know how you showed your stripes!

Today is !

Rare diseases impact 1 in 10 Americans. Everyone can play a role in helping to expand access to care and supporting research for people living with rare diseases.

Visit https://ow.ly/SIzW50Yl5qy to learn more and !

12/26/2025

The Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study (UNIFAI) is currently recruiting individuals of all ages and stages of Friedreich's ataxia (FA) at multiple global sites. The study will assess and evaluate clinical outcomes such as disease progression, symptom severity, and overall quality of life using data from medical history, health questionnaires, neurological and functional assessments, cardiac examinations, and laboratory studies.

For more information visit https://ow.ly/Y5Wq50WAVV7

Anyone considering participating in a clinical trial or study should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.

10/09/2025

Please join us for an afternoon of fun!

Jumpin’ For Jazz is a 501(c)(3) charity in Indianapolis that funds research and supports children with Friedreich’s Ataxia, an inherited neurological disease.
Help Jumpin’ for Jazz expand its mission and help as many as possible.

Join us for a fun-filled afternoon of live music featuring Rob Dixon and Sandy Williams, plus a silent auction, and great food, and community.
📅 Save the date: October 12
Secure your spot https://www.instantseats.com/index.cfm?fuseaction=home.event&eventID=0B86EF09-B0E4-88BD-A2EED9821F4C7016

07/19/2025

The "BRAVE" study is now open for enrollment at the Children's Hospital of the King's Daughter in Norfolk, VA and the Children's Hospital of Philadelphia in Philadelphia, PA. This study is sponsored by Biogen.

The study team is recruiting participants with a genetically confirmed FA diagnosis between the ages of 2-15 years. Participants must have no evidence of clinically significant heart disease and meet other study-specified eligibility criteria.

This global phase 3 study is investigating the safety and efficacy of omaveloxolone in children with FA. This is a one year placebo controlled trial where participants will take either omaveloxolone or placebo once a day. After one year, participants will be invited to enroll in an open label extension.

There are additional sites planned for this study both within and outside of the United States. FARA will continue to update the community as additional sites open.

Click the link for more information, eligibility requirements, and planned trial locations: https://ow.ly/ipAA50W9b4L. To learn more about the study and how to enroll, US residents can also contact the Biogen Patient Navigator at 1-877-223-3576 (access code 57078).

Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.

06/20/2025

Lexeo Therapeutics is conducting a natural history study called CLARITY-FA that is looking for individuals who have cardiomyopathy (heart muscle disease) associated with Friedreich ataxia (FA).

This study is recruiting participants over the age of 6 years with a genetically confirmed FA diagnosis. Participants must have evidence of cardiomyopathy and meet other study eligibility criteria.

The goal of this year-long study is to learn more about how heart disease develops and worsens in individuals with FA. This is a natural history study and participants will not receive any investigational study drug.

This is a global study that will have multiple sites across the US. Check the link below for currently recruiting sites and additional eligibility criteria.

Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.

Learn more about the study at https://ow.ly/hyIE50WbGRT

03/10/2025

Today, March 10, 2025, Design Therapeutics announced progress across its lead GeneTAC® programs including DT-216P2, a candidate being developed for the treatment of FA. A Phase 1 dosing study in healthy volunteers has been initiated in Australia to evaluate the safety and pharmacokinetics (PK) of single ascending doses of DT-216P2 via multiple routes of administration (intravenous infusion, subcutaneous infusion and subcutaneous injection). A Phase 1/2 multiple ascending dose (MAD) clinical trial to assess safety, PK and pharmacodynamics (PD) in FA patients is anticipated to begin in mid-2025.

Click here to read the full press release: https://ow.ly/cmjr50VeN6w

Click here to read more about the DT-216P2 program: https://ow.ly/GO2l50VeN6v

03/04/2025

Tissue donation is a unique gift. As one of our FA Accelerator researchers at Harvard Medical School, Dr. Syndi Barish, describes: "Tissue allows us to validate what we learn, which ultimately leads to better understanding and potential for better treatments." Learn more about how to pre-register to donate at: https://ow.ly/49pY50V81mF

03/04/2025

In a letter to the FA Community earlier this week, Biogen summarized key milestones for their FA program in 2024 including research highlights, commitment to children with FA, and expanding global access. Click here to read the letter: https://ow.ly/7R6V50V8Jr3

02/08/2025

Larimar Therapeutics is sponsoring a clinical trial to learn about the safety and tolerability of an investigational drug called nomlabofusp in children and adolescents with Friedreich's ataxia (FA). Nomlabofusp (previously called CTI-1601) is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia.

This study is currently recruiting U.S. participants at Uncommon Cures in Chevy Chase, Maryland. Participants must be 2 to 17 years old, have genetically confirmed FA, be able to traverse a distance of 25 feet with or without an assistive device, and meet other eligibility criteria.

Learn more about the eligibility requirements and how to enroll: https://buff.ly/3C7O4Ic

Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.

04/01/2024

SAVE THE DATE: The FA Research Reception is coming to Tacoma, WA!

Join us for this free in-person educational event on Saturday, August 24 from 1–5:30 p.m.

FA Research Receptions provide individuals and families affected by FA with up-to-date information about Friedreich’s ataxia research and an opportunity to meet others in the community.

FARA leadership and representatives from the FA research community will present on topics such as clinical management, the FA Drug Development Pipeline, and resources for clinical trial decision-making.

Date: Saturday, August 24, 2024
Time: 1–5:30 p.m.
Location: Hotel Murano
1320 Broadway, Tacoma, WA 98402

Registration, hotel block, and full agenda will be available soon!