Wednesday, April 24, 2024

Localized Changes in Dentate Nucleus Shape and Magnetic Susceptibility in Friedreich Ataxia

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Harding, I.H., Nur Karim, M.I., Selvadurai, L.P., Corben, L.A., Delatycki, M.B., Monti, S., Saccà, F., Georgiou-Karistianis, N., Cocozza, S....
Tuesday, April 23, 2024

Vesigen to Present New Preclinical Data on Engineered ARMMs Technology at 2024 ASGCT Annual Meeting

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Provided by Business Wire. Apr 22, 2024. Engineering ARMMs with Engagers to Direct Biodistribution to Specific Neurons as a Therapeutic Stra...

Lexeo Enters License Agreement for Friedreich Ataxia Treatment

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04.22.24. LEXEO Therapeutics, Inc., a clinical stage genetic medicine company, entered an in-license agreement with Cornell University to e...

Recent Advances in the Treatment Strategies of Friedreich’s Ataxia: A Review of Potential Drug Candidates and their Underlying Mechanisms

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S. Saini, Kumar Aman,A nil, Neha,Vijay, N. Ardra,Mangla, Bharti,Javed, Shamama,Kumar, Pankaj,Ahsan, Waquar, Recent Advances in the Treatmen...

The Role of Verbal Fluency in the Cerebellar Cognitive Affective Syndrome Scale in Friedreich Ataxia

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Corben LA, Blomfield E, Tai G, Bilal H, Harding IH, Georgiou-Karistianis N, Delatycki MB, Vogel AP. The Role of Verbal Fluency in the Cerebe...
Wednesday, April 17, 2024

Muscular Dystrophy Association and Friedreich’s Ataxia Research Alliance Announce Collaborative Research Grant Using Novel Gene Editing Technology to Address Root Cause of Friedreich’s Ataxia Disease

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NEW YORK, April 17, 2024 – The Muscular Dystrophy Association (MDA) and Friedreich’s Ataxia Research Alliance (FARA) announced today a colla...
Tuesday, April 16, 2024

LEXEO THERAPEUTICS GRANTED FDA FAST TRACK DESIGNATION FOR LX2006, AN AAV-BASED GENE THERAPY CANDIDATE FOR THE TREATMENT OF FRIEDREICH’S ATAXIA CARDIOMYOPATHY

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NEW YORK, April 16, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. today announced the U.S. Food and Drug Administration (FDA) has grante...

Pharmacotherapeutic strategies for Friedreich Ataxia: a review of the available data

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Gunther, K. and Lynch, D. R. (2024) ‘Pharmacotherapeutic strategies for Friedreich Ataxia: a review of the available data’, Expert Opinion o...
Thursday, April 11, 2024

Expression and processing of mature human frataxin after gene therapy in mice

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Rojsajjakul, T., Selvan, N., De, B. et al. Expression and processing of mature human frataxin after gene therapy in mice. Sci Rep 14, 8391 (...
Tuesday, April 9, 2024

Chaperone function in Fe–S protein biogenesis: Three possible scenarios

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Jaroslaw Marszalek, Elizabeth A. Craig, Marcin Pitek, Rafal Dutkiewicz, Chaperone function in Fe–S protein biogenesis: Three possible scenar...