Update on PPMD’s Risk/Benefit in Duchenne Therapies program

We were thrilled that 119 parents/guardians were willing and able to complete the entire treatment preferences survey. We are analyzing the data. We are doing the first part of the data analysis in-house, and the second part with our health economist collaborator. Our plan is to present this information to the FDA, to industry and clinical investigators, and back to you. We’ll also publish this data in a medical journal.

Share your story about Duchenne and potential treatments

We are still looking for people to share your story of what you want the FDA to know about Duchenne, potential treatments, and your family. We would especially like stories and comments from teens and adults with Duchenne, but we’d also like to hear from more parents and other relatives. We will continue collecting stories for a few more weeks. We plan to use these stories as part of our FDA strategy, and also share themes from the stories with biotech and pharmaceutical companies and clinicians.  (You get to choose how we use your information when you share your story.)

Share the story you would like the FDA to hear.

Looking Ahead

There is a lot of work still to do. We will be engaging you on a number of topics relevant to advancing our regulatory strategy throughout 2013. Though the final plan depends on the data from the survey and the “Share your story,” as well input from the FDA and other stakeholders, we anticipate asking the community to be in focus groups and take another survey. Your voice matters, and we cannot do any of this without your participation.  

We’re in this for the long haul. It’s not about one therapy or one trial—it’s about making the drug approval process work better to benefit our Duchenne community. We hope you’ll continue to partner with us along the way.


Holly Peay
Vice President, Education & Outreach

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