Monday, October 13, 2025

Analysis of a Modified Version of the Inventory of Non-Ataxia Signs Over 12 Years in Patients with Friedreich's Ataxia in the EFACTS Study

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Lischewski, S.A., Dogan, I., Giunti, P., Parkinson, M.H., Mariotti, C., Durr, A., Ewenczyk, C., Boesch, S., Nachbauer, W., Klopstock, T., St...
Thursday, October 9, 2025

LEXEO Message to the Friedreich Ataxia Community

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Message to the Friedreich Ataxia Community. October 7, 2025​. This morning, we were pleased to share positive interim clinical data for LX...

Frataxin depletion leads to decreased soma size and activation of AMPK metabolic pathway in dorsal root ganglia sensory neurons

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Frataxin depletion leads to decreased soma size and activation of AMPK metabolic pathway in dorsal root ganglia sensory neurons Olivier Gris...
Tuesday, October 7, 2025

Synthesis and Biological Profile of Omaveloxolone: The Cornerstone for Friedreich Ataxia Treatment

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Cordaro, M.; Neri, G.; Ansari, S.A.M.K.; Buccheri, R.; Scala, A.; Piperno, A. Synthesis and Biological Profile of Omaveloxolone: The Corners...

Lexeo Therapeutics Stock Rallies On Discussions With FDA To Expedite Friedreich’s Ataxia Drug Approval Process

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Published Oct 07, 2025. https://stocktwits.com  The company stated that data from a planned pivotal study will be pooled with data from the ...

Lexeo says FDA open to speedier approval of rare disease gene therapy

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Published Oct. 7, 2025. https://www.biopharmadive.com/  The agency will consider a submission that includes pooled data from ongoing studies...

A25-86 Omaveloxolone (Friedreich’s ataxia) – Benefit assessment according to §35a Social Code Book V

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Commission awarded on 01.07.2025 by the Federal Joint Committee (G-BA). Last updated 01.10.2025.  Result of dossier assessment: Added benefi...

Delphi study to elicit expert consensus around decision-making in the treatment of Friedreich ataxia

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Delphi study to elicit expert consensus around decision-making in the treatment of Friedreich ataxia. Front. Neurol.Sec. Movement Disorders ...
Monday, October 6, 2025

357PLong-term vatiquinone treatment slows Friedreich’s ataxia disease progression relative to FACOMS natural history

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357PLong-term vatiquinone treatment slows Friedreich’s ataxia disease progression relative to FACOMS natural history. Vagabov, A. et al. Neu...

234PLong-term use of omaveloxolone in patients with Friedreich ataxia: up to 5 years of natural history propensity score matching from the MOXIe OLE

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234PLong-term use of omaveloxolone in patients with Friedreich ataxia: up to 5 years of natural history propensity score matching from the M...