[Background]\ud Friedreich’s ataxia is an autosomal recessive, severely incapacitating disorder. There is little objective evidence regarding FRDA management. Abnormalities in the insulin/insulin-like growth factor 1 (IGF-1) system (IIS) signalling pathway were thought to play a role in the physiopathological processes of various neurodegenerative disorders, including spinocerebellar ataxias. The objective of the study was to test the safety, tolerability, and efficacy of therapy with IGF-1 in Friedreich’s ataxia (FRDA) patients in a clinical pilot study.
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机译：[背景] \ ud Friedreich的共济失调是一种常染色体隐性遗传病，严重无能。关于FRDA管理的客观证据很少。胰岛素/胰岛素样生长因子1（IGF-1）系统（IIS）信号传导途径的异常被认为在各种神经退行性疾病（包括脊髓小脑性共济失调）的生理病理过程中起作用。这项研究的目的是在一项临床试验研究中测试IGF-1治疗Friedreich共济失调（FRDA）患者的安全性，耐受性和疗效。
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