Friday, May 22, 2026

Slowing the curve: a single-arm meta-analysis of mFARS outcomes following omaveloxolone treatment in Friedreich ataxia

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Ali A, Cheema WA, Nisar F, Reyaz U, Saad M, Talha M, et al. Slowing the curve: a single-arm meta-analysis of mFARS outcomes following omavel...
Wednesday, May 20, 2026

Swinburne researcher discovers broccoli extract could halt incurable neurological disorder

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Newscorp Australia. May 19, 2026. Associate Professor Faith Kwa leads the Drug Discovery for Chronic Diseases Laboratory at Swinburne Unive...

New RESTORE-FA results show DT-216P2 improved balance and fatigue within 4 weeks, with tolerable safety.

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NeurologyLive®. May 19, 2026. DT-216P2 Shows Dose-Dependent Frataxin Increases and Clinical Improvements After 4 Weeks of IV Dosing in Fried...
Tuesday, May 19, 2026

Design Therapeutics Announces Four-Week IV Data from the RESTORE-FA Trial of DT-216P2 Demonstrating Clinical Improvements and Comprehensive Biomarker Activity in Friedreich Ataxia

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CARLSBAD, Calif., May 18, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developi...
Monday, May 18, 2026

140 Gene therapy for Friedreich ataxia cardiomyopathy: safety and preliminary assessment of efficacy

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2026 Annual Meeting of the American Society of Gene and Cell Therapy: ASGCT 2026 Annual Meeting Abstracts. Available online 8 May 2026, Vers...
Friday, May 15, 2026

69 Small‑Molecule–regulated RNA switch achieves therapeutically safe levels of Frataxin in mouse models of Friedreich’s Ataxia

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2026 Annual Meeting of the American Society of Gene and Cell Therapy: ASGCT 2026 Annual Meeting Abstracts. Available online 8 May 2026, Vers...

62 Development and Translation of PPL-001, an autologous, gene-edited CD34+ hematopoietic stem cell Therapy for Friedreich’s Ataxia

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2026 Annual Meeting of the American Society of Gene and Cell Therapy: ASGCT 2026 Annual Meeting Abstracts. Available online 8 May 2026, Vers...
Thursday, May 14, 2026

Neurophysiological assessment of disease severity in Friedreich’s Ataxia: a study of brainstem auditory and visual evoked potentials

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Simona Maccora, Umberto Quartetti, Salvatore Maria Lima, Nicasio Rini, Marco Cucchiara, Luisa Agnello, Caterina Maria Gambino, Filippo Brigh...

Experimental gene therapy raises hope for children with fatal neurological disease

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Brunel University of London. By Press Officen14 May 2026.  Scientists at Brunel University of London and University College London Great Orm...
Wednesday, May 13, 2026

Validation of circulating miR-323a-3p and miR-625-3p to classify hypertrophic cardiomyopathy in Friedreich’s ataxia

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Ibáñez-Cabellos, J.S., Baviera-Muñoz, R., Alemany-Perna, B. et al. Validation of circulating miR-323a-3p and miR-625-3p to classify hypertro...