Clinical Trial Readiness

Our Clinical Trial Readiness Program provides resources to support effective, patient-centered management of spinal muscular atrophy (SMA) clinical research and patient evaluation.

SMA Industry Collaboration

The Cure SMA Clinical Trial Readiness Program is an initiative developed under the SMA Industry Collaboration. Our goal is to alleviate challenges related to site capacity and enhance patient access to SMA clinical trials. The program offers resources for clinical research sites that seek to evaluate and optimize their readiness for SMA trials. Materials were created with input from the SMA patient community, health care providers, clinical researchers, and trial sponsors to highlight the evolving needs of SMA patients and families in an ever-changing landscape.

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Readiness Checklists

Cure SMA has created companion checklists to help clinical research professionals examine their preparedness for SMA clinical trials. Two checklists are available: one for trial sites and a second for clinical evaluators.

These were created with input from established research sites and sponsors of SMA clinical trials. For copies, please email [email protected].

Toolkits on SMA, Clinical Evaluation, and Clinical Research Coordination

Cure SMA has created a trio of toolkits for research teams. We have also compiled a list of resources from other organizations that may be helpful. We welcome feedback and ideas for topics to address. To receive information about future events and share your ideas, please email us at [email protected].


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.



Research coordinators play a pivotal role in the execution and management of SMA clinical trials, and their interactions with research participants can have an outsized impact on these individuals’ experiences in the trial, affecting recruitment, enrollment, and retention of participants. This toolkit reviews the roles and responsibilities of Clinical Research Coordinators in clinical trials, strategies and tactics to support trial management, and tips for working with individuals with SMA and their families.



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.



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 periodically hosts workshops for clinical research coordinators (CRCs), physical therapists (PTs), and clinical evaluators (CEs). To attend, individuals must register for the Research & Clinical Care Meeting.

Cure SMA also organizes webinars, which are recorded and available via Cure SMA’s Clinical Trial Readiness Program YouTube playlist.

SMArt Moves as a Resource

Developmental delays can be early signs of a serious medical condition. If something about a child’s development doesn’t feel quite right, don’t delay. SMArt Moves educates parents and healthcare providers about the signs of delayed milestones. With SMA, early diagnosis and treatment are key. Check out SMArt Moves!

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