7.9.13: Update from Congressional Briefing - TAKE ACTION

A big day for Duchenne – Take action to help make it even more impactful!


I had the pleasure to join other Duchenne advocates in Washington this week in an effort to bring attention to the need to accelerate treatments for Duchenne, highlighting eteplirsen as a prime example of a promising potential treatment for a subset of the Duchenne population.

A briefing was held at the Rayburn House Office Building, sponsored by Congressman Keating and organized by the Jett Foundation.  The patient voice was front and center, the room was filled to the max with members and staffers, and the message was: time is our enemy, doing nothing is doing harm, the patients are waiting.


Take Action


Please consider contacting your member of Congress and ask them to sign onto a letter of support to the FDA being circulated by Congressman Keating urging the FDA to move expeditiously with respect to a request for the approval of the accelerated marketing application for eteplirsen.

Ask to speak to the Health LA in their office, explain to them why this is important to you and your family, and if they have questions they can reach out to Sonya Spery in Congressman Keatings office by email sonya.spery@mail.house.gov or call 202-225-3111.




At the same time as the briefing, 20 miles away, PPMD and patient representatives held a meeting at the FDA with leadership that included Dr. Janet Woodcock. It was a productive and promising exchange regarding the urgency of getting Duchenne therapies to patients and what we’ve learned to date from patient families regarding benefit/risk.


Be sure to check out Pat’s summary of what came out of the meeting.

We continue to be the most effective when we work as one, consistent voice with one mission:

End Duchenne.

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