Wednesday, May 27, 2026

Xavier disease (Friedreich's ataxia variant) - Symptoms, Causes, Treatment & Prevention

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📅 Updated: April 2026. Xavier disease is a very rare, autosomal‑recessive neuro‑degenerative disorder that shares many clinical features wi...
Monday, May 25, 2026

Targeting competitive Fe–S regulation to treat Friedreich’s ataxia

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Campos J, Ferreira J. Targeting competitive Fe–S regulation to treat Friedreich’s ataxia. Trends in Pharmacological Sciences, 2026; 0 DOI: ...

A phosphorylated variant of the mast/stem cell growth factor receptor KIT is upregulated in dorsal root ganglia of Friedreich ataxia

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Koeppen AH, Mazurkiewicz JE, Feustel PJ, Pelech S, Sutter C, Fu Q, Lin Q. A phosphorylated variant of the mast/stem cell growth factor recep...

Psychological resilience in patients with Friedreich ataxia: a 6-year longitudinal analysis

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Ruth Eumann, Janna Krahe, Imis Dogan, Ana Sofia Costa, Jörg B Schulz, Stella A Lischewski, Kathrin Reetz; FACROSS Study Group. Psychologica...
Sunday, May 24, 2026

Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich’s ataxia models

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Pido-Lopez J, Moula S, Shaban E ... Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich’s at...
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...