Cure SMA Launches Benefit-Risk Survey to Assess Patient Perspective on Treatment Possibilities and Risks

Cure SMA is conducting a survey to understand our community’s perspectives on treatment possibilities and tolerance for the side effects of currently available and planned therapies. The results will help inform our future objectives and priorities as well as help us continue to bring the patient voice to regulatory authorities.

Some of you may recall having participated in a similar effort in 2017. Given the approval of additional therapeutics and many additional investigational drugs in clinical trials, we are seeking to readminister a very similar survey. Doing so, will allow us to capture shifts in patient and caregiver perspectives in response to the ever-evolving SMA landscape.

The data collected from this survey will allow us to better understand how individuals and families affected by SMA are currently thinking about risk assessments when coupled with specific benefits of a treatment. The learnings from this analysis will create a compelling case for the assessment and ultimate approval of new therapeutics in SMA. Findings will be compared to the benefit/risk analysis survey previously conducted in 2017.

We are encouraging every family in our community to complete this survey. An analysis of this survey will be shared with FDA as well as the SMA patient community. Help us to create a compelling case for the assessment and ultimate approval of new therapeutics to address unmet needs in SMA.

Who Can Participate in the Survey

  • Individuals with SMA age 18 or older.
  • Caregivers of individuals with SMA (all ages and types). Please note that caregivers must also be 18 years of age or older to participate.

How to Participate

Please use the link below and you will be directed to the survey. In this survey, you will be asked to rate the value of many treatment benefits and risks against each other. Given the diversity of presentation in SMA, the list of benefits and risks is a bit long as we want to make sure all SMA types and stages are represented in the findings. We appreciate your understanding when answering it, as this the required format for collecting benefit-risk information. The survey should take approximately 20 minutes to complete.

We want to hear from you! Take the survey now.


The survey will close on Friday, October 7th.

Take the survey!

For Assistance with Survey

Silicon Valley Research Group administering our survey. If you have any questions or problems accessing the survey or need assistance filling it out, please contact Mike Wasnock at [email protected].

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