PPMD is proud to host a Policy Forum on clinical trials of experiment agents on December 12, 2013 from 8.30AM until 4.30PM in Silver Springs, MD. We are especially pleased that key Duchenne-community stakeholders, including the U.S. Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER), and review divisions, are committed to the success of this day-long meeting. This meeting will be chaired by two well-regarded experts, Dr. Craig McDonald and Dr. Lee Sweeney.

"Optimizing Clinical Trials in Duchenne Muscular Dystrophy in the New Era of Improved Care Standards" is designed to create a draft guidance for the FDA that reflects the combined experience and wisdom of the Duchenne community. Modeled after the successful meeting held by our sister organization United Parent Project Muscular Dystrophy in London in the spring, the meeting will concentrate on four areas:

  • Unmet medical needs
  • Duchenne natural history in relation to clinical trial endpoints
  • Biomarkers and potential surrogate endpoints
  • Framing the draft guidance for optimizing Duchenne clinical trials



A different expert(s) will present on each topic. Following each presentation, comments from the Duchenne community will be welcome. Discussants must register to speak in advance and comments will be limited to three minutes. Our aim is to hear from all who have interest in the area as time allows.

If you would like to attend the meeting in person, please click here to register and to sign up to speak.

Share Your Story Online

This is your opportunity to Share Your Story with the FDA if you haven’t already. We realize traveling to Silver Springs may not be feasible for most of you. But by clicking here, you can submit your story to this ongoing PPMD initiative and we will present these stories to the FDA to help inform their decision-making process. The deadline for submitting your story is November 18, 2013.

Thank you for taking a few minutes to Share Your Story with the FDA so that they can hear more voices in this community.

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