The PPMD Town Hall on Clinical Trials, taking place on Monday, March 4th during PPMD’s 2024 Advocacy Conference in Washington, D.C., aims to provide a platform for families affected by Duchenne and Becker to share their experiences and insights, with a focus on past, present, and future clinical trials.

The primary goal is to hear from families and gather valuable input to inform PPMD’s advocacy efforts, particularly in interactions with the FDA on drug development. The meeting seeks to open a dialogue on regulatory advocacy priorities, focusing on what outcomes are meaningful to families.

Our agenda includes:

• Parent/Patient Panel, featuring a diverse group of parents and patients at different ages and stages and with a variety of clinical trial experiences, discussing challenges faced and expressing hopes for outcomes.
• Professional Panel focusing on Patient Preference and Clinical Trials, including a representative from FDA Center for Drug Evaluation and Research (CDER) as well as clinicians, covering FDA updates, issues in clinical trials, and potential solutions.
• The meeting will conclude by bringing together the insights gathered, discussing how clinical trials can be improved, and outlining recommendations.

Following the meeting, we plan to share a recap with the community and integrate learnings into our advocacy strategy moving forward as we interact with federal agencies (including FDA, NIH, DOD, and CDC). The ultimate aim is to enhance the clinical trial landscape for Duchenne and Becker families and contribute to policy strategies for improved outcomes.

Take Action

During March 4-5, over 120 PPMD advocates — people with Duchenne and Becker, parents, siblings, grandparents, and other family members and friends will be participating in meetings with Congressional offices, urging them to support vital efforts that will support continued progress for our community. Click here to take action and help reinforce our message to Congress from home.