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...
Tuesday, May 12, 2026

Pharmacological treatments for Friedreich ataxia ( Cochrane Database of Systematic Reviews 2026)

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Lyons S, Kearney M, Fahey MC, Janjal P, Pandolfo M, Patton P. Pharmacological treatments for Friedreich ataxia. Cochrane Database of Systema...

Transcription and Cohesin Direct Domain Boundary Spatial Positioning and are Linked to Friedreich's Ataxia

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Ashley Karnay, Ricardo Linares-Saldana, Qiaohong Wang, Zachary Gardner, Jialiu A. Liang, Garrett T. Santini, Krishna Kumar Haridhasapavalan,...
Sunday, May 10, 2026

442 FRATAXIN IS DOWNREGULATED IN COLONIC INFLAMMATION DISRUPTING EPITHELIAL HOMEOSTASIS

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Mo Wang, Xinqian Meng, Qiong Guo, Chunyan Peng, LEI WANG; 442 FRATAXIN IS DOWNREGULATED IN COLONIC INFLAMMATION DISRUPTING EPITHELIAL HOMEOS...

CGG, CAG, and GAA: Genome-wide comparison of the disease linked trinucleotide short tandem repeats

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Annear DJ, Vandeweyer G, Kooy RF. CGG, CAG, and GAA: Genome-wide comparison of the disease linked trinucleotide short tandem repeats. BMC Ge...

Comprehensive Review of Anesthetic Strategies for Patients With Neurodegenerative Diseases

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Grabarczyk Ł. Comprehensive Review of Anesthetic Strategies for Patients With Neurodegenerative Diseases. Med Sci Monit. 2026 Apr 11;32:e950...