Sunday, May 18, 2025

Modeling Friedreich's ataxia with Bergmann glia-enriched human cerebellar organoids

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Modeling Friedreich's ataxia with Bergmann glia-enriched human cerebellar organoids. Seungmi Ryu, Jason Inman, Hyenjong Hong, Vukasin M ...
Friday, May 16, 2025

Therapeutic combination of L-ascorbic acid, N-acetylcysteine, and dimethyl fumarate in Friedreich’s ataxia: insights from in vitro models

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Edzeamey, F. J., Ramchunder, Z., Valle Gómez, A., Ge, H., Marobbio, C. M. T., Pourzand, C., & Virmouni, S. A. (2025). Therapeutic combin...

Neuromagnetic Responses to Multimodal Stimuli in Friedreich’s Ataxia

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Elisa Visani, Laura Canafoglia, Lorenzo Nanetti, Davide Rossi Sebastiano, Dunja Duran, Paola Anversa, Deborah Bonfoco, Sara Dotta, Davide Ta...

Solid Biosciences Reports First Quarter 2025 Financial Results and Provides Business Updates

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GuruFocus News. 16/05/2025  The U.S. FDA has granted IND clearance for SGT-212, intended for treating Friedreich's Ataxia, with first pa...

Neurodegenerative and Metabolic Disease Challenges and Solutions at ASGCT 2025

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2025-05-15. ASGCT 2025. Neurodegenerative diseases like ALS, frontotemporal dementia, and Friedreich’s ataxia pose significant challenges wi...
Monday, May 12, 2025

Lexeo Therapeutics Reports First Quarter 2025 Financial Results and Operational Highlights

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NEW YORK, May 12, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. today provided business updates across its portfolio and reported first ...
Wednesday, May 7, 2025

PTC Therapeutics (PTCT) Q1 2025 Earnings Call

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May 6, 2025. Vatiquinone (Friedreich's Ataxia): FDA review proceeding without AdCom; confirmatory evidence from long-term data compared ...

Omaveloxolone for treating Friedreich’s ataxia in people 16 years and over (terminated appraisal)

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National Institute for Health and Care Excellence (NICE). 06 May 2025.  NICE is unable to make a recommendation on omaveloxolone (Skyclarys)...
Monday, May 5, 2025

Anti-gene oligonucleotides targeting Friedreich’s ataxia expanded GAA•TTC repeats increase Frataxin expression

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Negin Mozafari, Salomé Milagres, Tea Umek, Cristina S.J. Rocha, Claudia Marina Vargiu, Fiona Freyberger, Osama Saher, Marek Napierala, Jill ...

Hepatic Manifestations Following Gene Therapy

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Akash Roy, Shalini Bansal, Anand Kulkarni, K. Rajender Reddy, Hepatic Manifestations Following Gene Therapy, Gastro Hep Advances, 2025, 1006...