8:30 AM - 4:30 PM

AdvocacyDuchenne Policy Forum:

Optimizing Clinical Trials in Duchenne Muscular Dystrophy in the New Era of Improved Care Standards 


PPMD will host a Duchenne Policy Forum on clinical trials of experiment agents on December 12, 2013 from 8:30AM until 4:30PM at the Silver Spring DoubleTree by Hilton in Silver Spring, MD.

“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Clinical Trials in Duchenne Muscular Dystrophy in the New Era of Improved Care Standards” is designed to create a draft guidance for the FDA that reflects the combined experience and wisdom of the Duchenne community. 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 meeting.

Modeled after the successful meeting held by our sister organization United Parent Project Muscular Dystrophy in London in the spring, different expert(s) will present 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  • Framing the draft guidance for optimizing Duchenne clinical trials

Following each presentation, comments from the Duchenne community will be welcome. Speakers are requested to focus comments on the following:

  • The severity of the disease and impact on your life
  • The symptoms that matter most to you
  • Perspective on available therapies used to treat Duchenne

Comments will be limited to 2 minutes. Upon arriving at the conference, there will be a sign up available for all those interested in speaking.

Register to attend and/or speak

Space is limited, so you must register with PPMD in advance in order to attend and/or speak. Click the "Register Now" button below in order to register.

Unable to attend?

For those who are unable to attend, you can submit your story online (even if you wish to attend you can still submit a story). PPMD has been compiling stories for months, ensuring that the patient voice is heard, and sharing them with the FDA.

Hotel information

Silver Spring DoubleTree by Hilton 
8727 Colesville Road 
Silver Spring, MD 20910 
Phone: 301-563-3829

PPMD does not have a reserved room block or discounted rate for this event. View a list of area hotels.

Questions about the Policy Forum? Contact Ryan Fischer via email ryan@parentprojectmd.org 

This event is currently at capacity, please read our recent blog about how you can still participate. 

We're sorry, the deadline for registering for this event has passed.