Tuesday, April 27, 2021

AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases

›
CAMBRIDGE, MASS. AND SOMERSET, N.J. (PRWEB) APRIL 27, 2021  AavantiBio, a gene therapy company focused on transforming the lives of patients...
Sunday, April 18, 2021

Longitudinal structural brain changes in Friedreich ataxia depend on disease severity: the IMAGE-FRDA study

›
Selvadurai LP, Georgiou-Karistianis N, Shishegar R, Sheridan C, Egan GF, Delatycki MB, Harding IH, Corben LA.; J Neurol (2021). https://doi....
Wednesday, April 14, 2021

Remember friedreich ataxia even in a toddler with apparently isolated dilated (not hypertrophic!) cardiomyopathy: revisited

›
Anwar BABAN, Marianna CICENIA, Lorena TRAVAGLINI, Federica CALÍ, Gessica VASCO, Paola FRANCALANCI, Antonio NOVELLI, Rachele ADORISIO, Antoni...
Tuesday, April 13, 2021

The Cardioprotective Mechanism of Phenylaminoethyl Selenides (PAESe) Against Doxorubicin-Induced Cardiotoxicity Involves Frataxin

›
Fu Xiaoyu, Eggert Mathew, Yoo Sieun, Patel Nikhil, Zhong Juming, Steinke Ian, Govindarajulu Manoj, Turumtay Emine Akyuz, Mouli Shravanthi, P...
Monday, April 12, 2021

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

›
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

›
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

›
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

›
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

›
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

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