The Cure SMA Clinical Trial Readiness Program is designed to equip clinical trial sites with resources that support their ability to manage spinal muscular atrophy (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 offers tips for conducting clinical trials in SMA.

Readiness Evaluations for Research Sites and Clinical Evaluators

Cure SMA has created companion assessments that help research teams and clinical evaluators determine their preparedness for SMA clinical trials. Participating sites and clinical evaluators each complete a written questionnaire and meet virtually with Cure SMA regarding steps to optimize their site readiness. The programs are geared toward—but not limited to—prospective and new trial sites. To learn more, please complete this “Learn More and Download Toolkits” form.

Resources for Research Teams

To enhance site readiness and support trial conduct in SMA, Cure SMA has created a series of resources for research teams, in addition to identifying resources from other organizations that may be helpful to principal investigators (PIs), physical therapists/clinical evaluators (PTs/CEs), and clinical research coordinators (CRCs).

In recent years, major advancements have taken place in our understanding and ability to treat SMA. This toolkit discusses SMA, disease management and treatment approaches, steps involved in trial preparation and conduct, as well as things to consider when working in SMA. Finally, the toolkit offers an extensive list of resources prepared by other organizations and individuals to assist with protocol adherence. This information provides an essential foundation for work on SMA clinical trials. After completing this form, download links will appear below this toolkit description.

Research coordinators play a pivotal role in execution of SMA clinical trials, and their interactions with research participants can have an outsized impact on these individuals’ experiences, affecting recruitment, enrollment, and retention. This toolkit reviews CRC responsibilities, strategies, and tactics to support trial management, as well as tips on working with SMA-affected individuals and families. After completing this form, download links will appear below this toolkit description.

PTs/CEs play a fundamental role in the assessment of individuals with SMA. This toolkit was developed in collaboration with two PTs having significant experience in SMA. It is intended to help PTs/CEs—especially those new to SMA clinical trials—find productive ways to navigate challenges they may encounter in their work. The resource also includes appendices on additional resources, including seminal papers in SMA, manuals, and articles that delve further into SMA topics. After completing this form, download links will appear below this toolkit description.

Cure SMA has compiled information on numerous external resources from reputable sources that will be helpful to new and experienced research teams and, in particular, PTs and CRCs. These include training checklists, reading material, and information about online and in-person courses and workshops.

Workshops and Webinars

In conjunction with its Annual SMA Conference, Cure SMA holds workshops for CRCs and PTs/CEs. These events 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 & Clinical Care Meeting. Attendance is included with the cost of registration for the SMA Research & Clinical Care Meeting.

Cure SMA also organizes webinars, which are recorded and made available on this webpage. See below for available recordings.

Scholarship Program

Cure SMA has launched a new scholarship program to support the professional development of PIs, PTs, and CRCs involved in SMA clinical trials. This program is being launched with the support of the SMA Industry Collaboration and supports participation in virtual and in-person professional development activities relevant to the conduct of SMA clinical trials. Award amounts vary, and applications will be accepted on a rolling basis, as funds remain available.

Click here for more details

Click here to apply

Get In Touch and Request More Information

We welcome feedback on this program, as well as ideas for activities and topics to address. 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.

If you use our toolkits, we welcome feedback via the links below. Your input allows Cure SMA to optimize future versions to best meet your needs.

» Provide Feedback on the Trial Readiness Toolkit

» Provide Feedback on the Clinical Research Coordinator Toolkit

» Provide Feedback on the Physical Therapist and Clinical Evaluators Toolkit