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...

Friedreich Ataxia and Related Diabetes: Therapeutic Approach Targeting Mitochondrial Dysfunction

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Pichakacheri Sureshkumar, Sidharth S Kumar, Johny Cheriyan, Asif Masood, Friedreich Ataxia and Related Diabetes: Therapeutic Approach Target...

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

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A. Vagabov, J. Cherry, A. Duqette, M. França, S. Perlman, A. Durr, E. Bertini, K. Mathews, L. Schöls, A. Fournier, M. Delatycki, S. Subramon...
Saturday, October 4, 2025

Emerging therapies for Friedreich Ataxia and the prospect of future combination treatments

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Lees, J. G., Li, L., & Lim, S. Y. (2025). Emerging therapies for Friedreich Ataxia and the prospect of future combination treatments. Fu...