Tuesday, December 30, 2025

Development of a secretable frataxin for enhanced efficacy in treating Friedreich’s Ataxia

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Daniel M. DuBreuil, Michael Fleming, Yashvi Parikh, Mikaela Woo, Jie Bu, Swathi Ayloo, Ingeborg M. Langohr, Dinesh S. Bangari, Christian Mue...

Using a discrete choice experiments to explore societal preferences for valuing new drugs for rare diseases

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Vargas, C., Goodall, S., Street, D.J. et al. Using a discrete choice experiments to explore societal preferences for valuing new drugs for r...
Monday, December 29, 2025

Progressive scoliosis in a pediatric patient with cerebellar ataxia: surgical challenges and literature review

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Asunis E, Vitulli F, Nicotra R, Rubino A, Cicala D, Cinalli G, Colella G. Progressive scoliosis in a pediatric patient with cerebellar ataxi...

Digital Gait Measures Discriminate People with Friedreich's Ataxia from Healthy Controls

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Casey HL, Shah VV, Muzyka D, McNames J, El-Gohary M, Sowalsky K, Safarpour D, Carlson-Kuhta P, Schmahmann JD, Rosenthal LS, Perlman S, Rumme...
Sunday, December 28, 2025

Neuropathology of Friedreich ataxia and its links to metabolic pathways

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Mercado-Ayón E, Lazaropoulos MP, Mercado-Ayón Y, Lynch DR. Neuropathology of Friedreich ataxia and its links to metabolic pathways. Neurodeg...

Unrecognized high prevalence of expanded composite repeats in Friedreich ataxia

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Devore MC, Lam C, Wiley G, Park CC, Lynch DR, Bidichandani SI. Unrecognized high prevalence of expanded composite repeats in Friedreich atax...
Friday, December 26, 2025

(Italy). The first treatment (Skyclarys) for Friedreich's ataxia will be reimbursed by the National Health Service

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At its meeting on 15 December, the AIFA Board of Directors gave the green light to the reimbursement of Skyclarys (omaveloxolone), an orphan...
Tuesday, December 23, 2025

Respiratory Function in Friedreich's Ataxia

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Viana CF, Jaques CS, Bezerra MLE, Barsottini OGP, Pedroso JL. Respiratory Function in Friedreich's Ataxia. Mov Disord. 2025 Dec 19. doi:...

Ferroptosis is a novel pathogenic mechanism of FDXR-related disease via disruption of the NRF2 pathway

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Campbell, T., Slone, J., Vu, J. et al. Ferroptosis is a novel pathogenic mechanism of FDXR-related disease via disruption of the NRF2 pathwa...
Saturday, December 20, 2025

Targeting Friedreich Ataxia: A Sustainable Path to Safer and Smarter Therapeutics Through Integrated Docking and Toxicology

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H. B. Attel, R. P, A. K, B. S, Shivandappa and S. Manokaran, "Targeting Friedreich Ataxia: A Sustainable Path to Safer and Smarter Ther...