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LAST CALL: Phase 1 Survey - Risk/Benefit in Duchenne Therapies

By now you’ve probably heard about PPMD’s Risk/Benefit in Duchenne Therapies program, which aims to inform the FDA and other government agencies, biopharmaceutical companies, and others about the treatment priorities and risk tolerance of the Duchenne community. We’ve had a great response to our first survey, with more than 90 participants so far.

Survey - Last Call! 

This is our last call for participants for the survey. If you have already done the survey, thanks so much! If you haven’t done the survey, or are in the middle of completing it, please do so before 9:00 PM Eastern time on Wednesday, Feb 20th.

We especially need parents OR guardians of:

  • Very young children, especially age 4 and under
  • Children who are able to walk some, but who are having a lot of difficulty
  • Adults who have lost the ability to power their own wheelchair

 

Take the treatment priorities survey now.

 

Share Your Story with the FDA - Still Ongoing

People with Duchenne and Family Members: Share your story of what you want the FDA to know about Duchenne, potential treatments, and your family. We will continue collecting these stories for a few more weeks.

 

Next Steps

Through 2013, we will continue to engage our community about risk tolerance, benefit preferences, and treatment priorities. The next phases will include getting additional information about treatment preferences and risk tolerance from more of the community.

 

We will keep you informed as the project moves forward!



Holly Peay
Vice President, Education & Outreach

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Tags: PPMD Blog, Staff, fda, risk/benefit

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Comment by Marian Lamberson on February 19, 2013 at 3:39pm

tried to take the survey, but not eligible in Canada.  Good luck!!

 

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