Wednesday, October 22, 2025

Exploring the pleiotropic effects of lncRNA in different repeat expansion disorders

›
Soumalya Das, Shubhi Khandelwal, Sakshi Shukla, Amit Kumar, Exploring the pleiotropic effects of lncRNA in different repeat expansion disord...
Monday, October 20, 2025

A Probabilistic Deep Ensemble Framework for the Objective Assessment of Friedreich Ataxia

›
Maneesha Randeniya, Kanishka Ranaweera, Thang Ngo, et al. A Probabilistic Deep Ensemble Framework for the Objective Assessment of Friedreich...

Chapter 23 - Omaveloxolone: a nuclear factor erythroid 2-related factor 2 activator for Friedreich’s ataxia

›
Ziquan Zhao, Junjie Wang, Mengchen Lu, Qidong You, Zhengyu Jiang, Chapter 23 - Omaveloxolone: a nuclear factor erythroid 2-related factor 2 ...
Thursday, October 16, 2025

Targeting rare splicing defects: Antisense oligonucleotides offer a therapeutic strategy in FRDA

›
Targeting rare splicing defects: Antisense oligonucleotides offer a therapeutic strategy in FRDA, Kerkhof, Laurie M.C. et al., Molecular The...
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

›
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

›
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

›
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

›
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

›
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

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