Friedreich's Ataxia Insights

This week's must-know community updates, latest research & events

March 19, 2025 • Estimated Reading Time: 1 minute

Introduction to Gene Therapy - out now!

We're thrilled to announce the launch of the first guide in our gene & cell therapy series.

Read it for free here:

www.allmyhealth.io/reports

Stay tuned for next week when we will launch the next report in the series.

Would you like to sponsor the next report in the series?

Enquire here

Latest Research

The TRACK-FA study, as detailed by Georgiou-Karistianis et al. (2025), is a significant effort to understand the progression of changes in the brain and spinal cord in individuals with Friedreich's ataxia. The study is supported by grants from various organizations, including the Friedreich's Ataxia Research Alliance (FARA) and pharmaceutical companies. The funding bodies are involved in study oversight and design, and some authors from these organizations contributed to the manuscript preparation.

Monash University serves as the coordinating site for this multi-site study, which includes universities and hospitals across the globe. The study aims to provide a comprehensive view of the disease's impact on the central nervous system, which could be crucial for future therapeutic strategies. The involvement of these institutions and companies underscores the collaborative nature of the research and the importance of tracking the natural history of Friedreich's ataxia.

Community News

National Ataxia FoundationMar 12, 2025

Did you know? The cerebellum is the part of the brain responsible for movement coordination in the body. Ataxia symptoms occur when the cerebellum is not functioning properly. Cerebellar dysfunction can be caused genetically like in the SCAs or by physical injury. #AtaxiaAwareness

As part of @danafoundation #BrainAwarenessWeek, we'll be sharing facts about Ataxia all week long! Share, like, and comment on our posts to help create awareness for #Ataxia!

Cure FAMar 17, 2025

Biogen Canada Inc. announced today, March 17, 2025, that Health Canada has approved SKYCLARYS™ (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in patients 16 years of age and older.

Montreal-based neurologist and researcher, Dr. Massimo Pandolfo, said, “The approval of SKYCLARYS is a significant advancement, offering the first therapy to go beyond managing symptoms and to targeting disease progression. While not a cure, this development represents meaningful progress in treatment options and brings renewed hope to patients and their families.”

"The approval of SKYCLARYS brings long-awaited hope to the patient community. It is a significant milestone for Canadians affected by Friedreich’s ataxia and one our founder Claude St Jean would have been thrilled about. This therapy marks a crucial development in addressing a condition that severely impacts the lives of those living with it and their families," said François-Olivier Théberge, General Manager, Ataxia Canada.

Read the full press release here: https://ow.ly/LeyZ50Vj6nr