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- W4378678082 startingPage "291" @default.
- W4378678082 abstract "Spinocerebellar ataxias (SCAs) are a group of dominantly inherited disorders with progressive cerebellar dysfunction. Although there are no Food and Drug Administration-approved therapies in the United States for SCAs, the efforts in the past decades have helped us gain an understanding of the pathomechanisms and disease progression, especially with cytosine-adenine-guanine (CAG) expansion SCAs. This has set the stage for the development of symptomatic or disease-modifying therapies. However, when designing clinical trials, it is important to choose suitable clinical rating scales to monitor disease progression and response to therapeutic interventions. In addition, studies need to incorporate appropriate biomarkers that can be used to test for target engagement. This chapter will review the rating scales and recent advances of biomarkers, focusing on CAG-repeat SCAs. Understanding these available tools will facilitate the design of clinical trials to find therapies for SCAs." @default.
- W4378678082 created "2023-05-30" @default.
- W4378678082 creator A5009039130 @default.
- W4378678082 creator A5040931027 @default.
- W4378678082 date "2023-01-01" @default.
- W4378678082 modified "2023-10-16" @default.
- W4378678082 title "Therapy Development for Spinocerebellar Ataxia: Rating Scales and Biomarkers" @default.
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