期刊
NEUROLOGIA I NEUROCHIRURGIA POLSKA
卷 52, 期 2, 页码 129-139出版社
ELSEVIER URBAN & PARTNER SP Z O O
DOI: 10.1016/j.pjnns.2018.02.003
关键词
Friedreich ataxia; Therapy; Rehabilitation; Gene therapy
资金
- Friedreich Ataxia Research Association (Australasia)
- Friedreich's Ataxia Research Alliance (United States of America)
- Murdoch Children's Research Institute
- NHMRC Early Career Fellowship
- Medical Research Future Fund Next Generation Researchers Fellowship
Friedreich ataxia (FRDA) is a progressive neurological disorder affecting approximately 1 in 29,000 individuals of European descent. At present, there is no approved pharmacological treatment for this condition however research into treatment of FRDA has advanced considerably over the last two decades since the genetic cause was identified. Current proposed treatment strategies include decreasing oxidative stress, increasing cellular frataxin, improving mitochondrial function as well as modulating frataxin controlled metabolic pathways. Genetic and cell based therapies also hold great promise. Finally, physical therapies are being explored as a means of maximising function in those affected by FRDA. (c) 2018 Polish Neurological Society. Published by Elsevier Sp. z o.o. All rights reserved.
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