The Cure SMA Clinical Trial Readiness Program is designed to equip clinical trial sites with resources that support their ability to manage SMA clinical trials in an effective and patient-centered way. The program includes voluntary educational evaluations to assess a site’s preparedness for SMA trials, as well as print resources and periodic events to help sites and teams learn about SMA and tips for conducting clinical trials in SMA.

Site and Physical Therapist/Clinical Evaluator Readiness Programs

The site and clinical evaluator readiness programs are companion programs that, respectively, help principal investigators and clinical evaluators determine their preparedness for SMA clinical trials. When a site or individual clinical evaluator takes part in these programs, they complete a written questionnaire followed by a discussion with Cure SMA regarding their questionnaire and steps that could be taken to optimize their site or individual readiness. To learn more about these programs, please complete this “Learn More and Download Toolkits” form.

Best Practices Toolkits

Cure SMA has created a series of three complementary toolkits to help research teams and clinical sites prepare for and conduct SMA clinical trials. You may download the toolkits by completing this “Learn More and Download Toolkits” form. Once you have completed the form, download links will appear below each toolkit description. If you use any of these toolkits, we encourage you to provide feedback via the links below each toolkit description. This will allow Cure SMA to assess the utility of the toolkits and how to best optimize future versions to best meet your needs. We greatly appreciate your feedback.

Toolkit 1: The Basics of Spinal Muscular Atrophy and the Effective Conduct of Clinical Trials in SMA

In recent years, major advancements have taken place in our understanding and ability to treat SMA. Shifts include a better understanding of natural history, new approved and investigational drugs, and more widespread adoption of newborn screening. This toolkit focuses on SMA as a disease and the landscape for disease management and treatment options, providing research teams with foundational knowledge and vocabulary to aid them in their work. It also discusses steps involved in trial preparation and conduct, issues that may arise in SMA trials, and things to consider in your day-to-day work. Finally, the toolkit offers an extensive list of resources prepared by other organizations and individuals to assist with protocol adherence (e.g., tools and templates for tracking study related procedures; lists of essential documents for before, during, and after a visit; a glossary of commonly used terms; and much more!).

» If you have already downloaded, please Provide Feedback on the Trial Readiness Toolkit

Toolkit 2: Best Practices for Clinical Research Coordinators in SMA

Clinical research coordinators (CRCs, also known as Study Coordinators) play a pivotal role in successful execution of an SMA clinical trial, in that their interactions with research participants and their families can have an outsized impact on these individuals’ experiences throughout the trial process, and determine how effective sites are at recruitment, enrollment, and retention. This toolkit, tailored specifically to the role of the CRC, includes a brief overview of the responsibilities of a CRC when coordinating any clinical trial, as well as information on steps to take when preparing for an SMA trial. The majority of the toolkit focuses on key stages and aspects of SMA clinical trials, with discussion of strategies and tactics to support trial management, and specific challenges that CRCs may encounter in the context of SMA clinical trials.

» If you have already downloaded, please Provide Feedback on the Clinical Research Coordinator Toolkit

Toolkit 3: Best Practices for Physical Therapists and Clinical Evaluators in SMA

Physical therapists/clinical evaluators (PTs/CEs) play a fundamental role in the assessment of individuals with SMA, which may involve impairment-based measures, as well as capacity and/or performance-based measures. To support PTs/CEs involved in SMA trials, Cure SMA, in collaboration with two PTs with significant experience in SMA and SMA clinical trials, has developed recommendations to promote the most effective conduct in clinical trials. These best practices are intended to help PTs/CEs – especially those new to SMA clinical trials – understand challenges they may encounter and find productive ways to navigate these challenges. Also included in this document is a set of appendices with links to additional resources, including seminal papers in SMA, manuals, and articles that delve more deeply into SMA issues.

» If you have already downloaded, please Provide Feedback on the Physical Therapist and Clinical Evaluators Toolkit


In conjunction with its Annual SMA Conference, Cure SMA holds workshops for clinical research coordinators as well as physical therapists and clinical evaluators. These are half-day to day-long events that focus specifically on the roles, responsibilities, and interests of their target audiences. To attend the workshops, individuals must register for the Annual International SMA Research and Clinical Care Meeting. Workshop attendance is included with the cost of registration for the Research and Clinical Care Meeting.


Cure SMA occasionally organizes webinars as part of the Clinical Trial Readiness Program, to expand the reach of its activities to sites across the U.S. These webinars will be recorded and made available on this webpage. Check out our webinar from December 2019 below.

Get In Touch, Share Your Ideas, and Request More Information

We welcome feedback on this program, and ideas for activities and topics that might be taken up in the context of the program. To receive information about future events and share your ideas, please email us at [email protected] or complete our “Learn More and Download Toolkits” form.