Patient experience data is a critical tool for advancing drug development in Duchenne and for regulatory decision making.

Steroids have been part of care standards for over two decades, however, there is a lack of data measuring how the community thinks and feels about taking steroids and about your overall experiences.

With this gap in data, PPMD has launched a first of its kind Duchenne Steroid Patient Experience Survey.

About the Survey:

Parent Project Muscular Dystrophy has partnered with RTI International to explore decision-making around steroids and patient experiences using steroids. Data from this study will published so that everyone can learn from the results. Your responses will remain confidential.

Everyone who completes this survey will be entered into a raffle to win one of FIVE $50 Amazon gift cards! The drawing will occur once recruitment is completed.

*For those who have already taken the survey through an email sent via The Duchenne Registry, thank you! You have been entered into the raffle. 

Study inclusion:

• Adults (age 18 and up) who are using steroids
• Parents of children (any age) who are using steroids

We are recruiting approximately 250 participants for this study. The survey will be confidential, and all questions are voluntary. The survey will take no more than 30 minutes to complete.

How to participate: 

If you are interested in participating in this study, please click the correct link below to determine if you are eligible:

Adult with
Duchenne (18+) > Parent/Caregiver >

If you have questions about participation and want to learn more, please contact Ryan Fischer at ryan@parentprojectmd.org or Brian Denger at brian@parentprojectmd.org

This study is sponsored in part by Catabasis Pharmaceuticals, Mallinckrodt Pharmaceuticals, PTC Therapeutics, and Santhera Pharmaceuticals. We thank the companies for their willingness to support a pre-competitive collaborative study to advance our understanding of patient experiences.