Monday, April 12, 2021

Diseases Costs and Impact of the Caring Role on Informal Carers of Children with Neuromuscular Disease

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Rodríguez, A.A.; Martínez, Ó.; Amayra, I.; López-Paz, J.F.; Al-Rashaida, M.; Lázaro, E.; Caballero, P.; Pérez, M.; Berrocoso, S.; García, M....
Friday, April 9, 2021

A Phase 2a investigator-initiated open-label clinical trial assessing elamipretide

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BOSTON, April 6, 2021 /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the dis...
Thursday, April 8, 2021

Healx launches partnership with Ataxia UK and FARA to find treatments for rare neurodegenerative condition

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Cambridge UK – 7 April 2021 – Healx, the AI-powered, patient-inspired technology company accelerating the discovery and development of rare ...
Wednesday, April 7, 2021

Biochemical alterations precede neurobehavioral deficits in a novel mouse model of Friedreich ataxia

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Marta Medina-Carbonero, Arabela Sanz-Alcazar, Elena Britti, Fabien Delaspre, Elisa Cabiscol, Joaquim Ros, Jordi Tamarit; bioRxiv 2021.04.05....
Thursday, April 1, 2021

Synthesis of 5‐[(1H‐indol‐3‐yl)methyl]‐1,3,4‐oxadiazole‐2(3H)‐thiones and their protective activity against oxidative stress

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škauskienė, M., Kadlecová, A., Voller, J., Janovská, L., Malinauskienė, V., Žukauskaitė, A., Šačkus, A., Arch. Pharm. 2021, e2100001. doi:10...
Wednesday, March 31, 2021

Lessons for clinical trial design in Friedreich's ataxia

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Masha G Savelieff Eva L Feldman The Lancet Neurology; Published:March 23, 2021DOI:https://doi.org/10.1016/S1474-4422(21)00064-8 Friedreic...
Tuesday, March 30, 2021

Rare-disease researchers face unique obstacles: Minoryx

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29-Mar-2021 By Jenni Spinner A representative of the rare-disease specialist firm outlines the unique obstacles of orphan CNS disease resea...

Mapa epidemiológico transversal de las ataxias y paraparesias espásticas hereditarias en EspañaEpidemiology of ataxia and hereditary spastic paraplegia in Spain: a cross-sectional study

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G. Ortega Suero, M.J. Abenza Abildúa, C. Serrano Munuera, I. Rouco Axpe, F.J. Arpa Gutiérrez, A.D. Adarmes Gómez, F.J. Rodríguez de Rivera, ...
Monday, March 29, 2021

Pre-clinical left ventricular myocardial remodeling in patients with Friedreich's ataxia: A cardiac MRI study

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Takazaki KAG, Quinaglia T, Venancio TD, Martinez ARM, Shah RV, Neilan TG, Jerosch-Herold M, Coelho-Filho OR, França MC Jr.; PLoS One. 2021 M...
Saturday, March 27, 2021

Quantitative assessment of Friedreich Ataxia via self-drinking activity

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Ragil Krishna, Pubudu N Pathirana, Malcolm Horne, Louise Corben, David Szmulewicz; IEEE J Biomed Health Inform. 2021 Mar 25;PP. doi: 10.1109...