Fig. 1.

Establishment of the Cure SMA Clinical Trial Readiness Program. Establishment of this program included three phases. Prior to creating the Clinical Trial Readiness Program, Cure SMA sought insight into the need for new clinical trial sites, existing clinical trial and care readiness programs, and factors important to trial management in SMA. Findings from this assessment informed the scope and focus of the trial readiness program, which was developed in 2018 and – in its pilot phase – included three components: a virtual site evaluation, a program for physical therapists and clinical evaluators, and a clinical trial readiness toolkit. The program was launched in the spring of 2018, and has since been refined and expanded to include best practices workshops and toolkits for clinical research coordinators as well as physical therapists and clinical evaluators