ADD YOUR VOICE: Participate in PPMD’s Gene Therapy Survey


Patient-Focused Drug Development in action


Companies developing gene therapies, the FDA, and other stakeholders are interested in the treatment priorities of the Duchenne community.  Because of this, PPMD has collaborated with RTI International, with financial support from Solid Biosciences and Pfizer, to launch a survey through Duchenne Connect. 


The goal of the survey is to learn about your thoughts and preferences about using gene therapy technologies as a possible treatment for Duchenne.
 Participating in the survey ensures your voice, your perspective, is included in drug development so that we can better communicate to companies and regulators about how you think and feel regarding emerging gene therapies. The more people that take the survey the better understanding we have of community priorities and perspectives regarding gene therapies. Your participation in research like this, gives you a seat at the table, providing real meaning to Patient-Focused Drug Development.

How to access the survey


This survey is online and anonymous meaning that no one at RTI or PPMD knows who took the survey or what they said. The only way to take this survey is by being registered with DuchenneConnect. You should have received an email last week asking you to participate.

Who can take the survey?

  • Caregivers of someone diagnosed with Duchenne

  • Adults with Duchenne, 18 and older


Again, you should have received an email from DuchenneConnect last week (if you are registered) asking you to participate.


Please forward the email you received from DuchenneConnect to your child if they are 18 years or older, as we are in need of responses from adults with Duchenne along with caregivers.


You can both take the survey separately.

How long will it take?


The survey takes about 45 minutes. Unfortunately, there is no compensation for completing the survey. However, the data collected is critical to advancing patient-focused drug development in gene therapy.


There are no right or wrong answers; we are looking for your opinions. 


What if I started the survey but didn’t finish it?


If you use the same computer, you should be able to restart where you left off by clicking on the same link from the email.


In order for the data to be used, the surveys must be complete so please be sure your voice is included!


If you have questions or concerns, contact Ryan Fischer at PPMD ryan@parentprojectmd.org or Holly Peay at RTI hpeay@rti.org.

Thank you to everyone who has already completed the survey!

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