Friedreich's Ataxia Insights

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

March 26, 2025 • Estimated Reading Time: 1 minute

New Report: Introduction to Cell Therapy

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Latest Research

Recent research has explored the potential of lipid nanoparticle (LNP) mediated mRNA delivery as a therapeutic strategy for neurodegenerative diseases, including Friedreich's ataxia. This approach aims to overcome challenges in treating these conditions by delivering genetic material directly to cells, which could lead to new treatments Moosavi et al. (2025). This method involves using tiny particles to carry mRNA into cells, potentially offering a way to address the genetic causes of diseases like Friedreich's ataxia.

The use of LNP-mediated mRNA delivery represents a promising avenue for developing treatments that target the underlying genetic issues in neurodegenerative diseases. While the specifics of how this technology might be applied to Friedreich's ataxia are not detailed in the provided information, the approach generally involves delivering genetic instructions to cells to produce specific proteins or modify gene expression. This could potentially lead to therapies that improve symptoms or slow disease progression in conditions like Friedreich's ataxia.

Clinical Trials

This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:

View the full list of clinical trials →

Community News

National Ataxia FoundationMar 20, 2025

Today is CACNA1A Awareness Day which was introduced by the Cacna1a Foundation in 2020! They chose the 19th because the CACNA1A gene is located on Chromosome 19. Various types of Ataxia are caused by different mutations on the CACNA1A gene such as Spinocerebellar Ataxia type 6 (SCA6) and Episodic Ataxia type 2 (EA2).

Learn more about SCA6 here: https://www.ataxia.org/sca-6/

Learn more about Episodic Ataxia here*: https://www.ataxia.org/ea/

*(This page includes information about Episodic Ataxias in general rather than focusing specifically on EA2)

Ataxie Ataxia CanadaMar 20, 2025

Did you know that Sinem Kara 🎉, one of the four finalists of Big Brother Celebrity Québec, has chosen the Ataxia Canada Foundation 💙 as the beneficiary of the $25,000 grant 💰 if she wins the competition?! 🤩👏

Let's wish her the best of luck! 🍀 And most importantly, the best strategy! 😉

Oh! And if you could take just 5 seconds to film yourself saying "Thank you!" 🎥, we’re putting together a beautiful collective thank-you video for her! 💙 Just send it to us via Messenger, and we’ll take care of the editing! 🎬😊

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Saviez-vous que Sinem Kara 🎉, l'une des quatre finalistes de Big Brother Célébrités Québec, a choisi la Fondation Ataxie Canada 💙 comme bénéficiaire de la bourse de 25 000 $ 💰 si elle remporte la compétition ?! 🤩👏

Souhaitons-lui la meilleure des chances ! 🍀 Et surtout, la meilleure stratégie ! 😉

Oh ! Et si vous pouviez prendre juste 5 secondes pour vous filmer en disant "Merci !" 🎥, nous préparons une belle vidéo collective de remerciements pour elle ! 💙 Il suffit de nous l'envoyer via Messenger, et nous nous occuperons du montage ! 🎬😊

Cure FAMar 24, 2025

In a press release today, March 24, 2025, Larimar Therapeutics shared several clinical and regulatory updates on their nomlabofusp program in FA.

Larimar reported that the Open Label Extension Study in adults continues to enroll, and that the adolescent cohort of the Pediatric PK run-in study is anticipated to complete dosing at the end of this month.

Larimar also obtained feedback from both the FDA and EMA on the global Phase 3 study protocol and reported that they are on track to initiate this study by mid-2025, with potential sites in the U.S., Europe, U.K., Canada, and Australia.

As part of the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program, Larimar shared that they have had more frequent interactions with the FDA, which have been informative as the company plans its Biologics License Application (BLA) submission.

To read more about these updates, click here: https://ow.ly/2p0o50VngCj