Fig. 2.

Improving clinical trial structure in SCAs. An outline of methods that can be used to increase the statistical significance of clinical trials in rare diseases such as SCA1 is provided. Because achieving statistical significance in clinical trials for SCAs is difficult, we must rethink the design of traditional trials. For example, when the treatment target is shared with other SCAs, SCA1 may be grouped with other SCAs. By creative reorganization and addressing symptoms as well as underlying disease processes, we can more easily bring compounds from bench to bedside