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...
Friday, April 5, 2024

AAV gene therapy to treat Friedreich’s ataxia cardiomyopathy

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Ferreira, J. AAV gene therapy to treat Friedreich’s ataxia cardiomyopathy. Lab Anim 53, 86 (2024). doi:10.1038/s41684-024-01351-0

NeuroVoices: Francesco Saccà, MD, PhD, on Crossing Over Dimethyl Fumarate in Friedriech Ataxia

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NeurologyLive. April 3, 2024. We completed the enrollment in February. All 40 patients were enrolled. We are expecting to have the last pat...

Approval of omaveloxolone for Friedreich ataxia

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Boesch, S., Indelicato, E. Approval of omaveloxolone for Friedreich ataxia. Nat Rev Neurol (2024). doi:10.1038/s41582-024-00957-9  The recen...
Tuesday, April 2, 2024

Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD study

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Rummey C, Perlman S, Subramony SH, Farmer J, Lynch DR. Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD stud...
Friday, March 29, 2024

Therapeutic Potential of Dimethyl Fumarate to Treat Friedreich Ataxia: Francesco Saccà, MD, PhD

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NeurologyLive. March 28, 2024. Fracesco Saccà, MD, PhD In an interview with NeurologyLive®, Saccà discussed the idea behind the study and th...