Update from PPMD's One Voice Advocacy Summit - 1st Panel

Pat Furlong opened the One Voice Summit for Duchenne Muscular Dystrophy welcoming attendees.  She provided background of the MD CARE Act and the accomplishments over the past ten years including the Muscular Dystrophy Coordinating Committee’s Action Plan for muscular dystrophy.  Pat spoke about those who helped initiate Parent Project Muscular Dystrophy’s entry into the advocacy including meetings and advice provided related to Senators Arlen Spector and Roger Wicker, Joel Wood and Lee Sweeney.  Pat spoke about the changes the community has seen such as the $192 million spent on DMD by the federal government, reducing the time to receive a genetic test from 3 to 6 months to a matter of days, a paucity of studies related to DMD to over 50 listed on the ClinicalTrials.gov website, global patient registries and the interest from biotechs and big pharma in DMD.  Pat explained the format for the meeting with three panels focusing on aspects of the MDC Action Plan that would eventually be published in Roll Call magazine which all members of congress and their staff read.

 

The first panel for the One Voice Summit featured six parents with their thoughts of Duchenne Muscular Dystrophy related the Muscular Dystrophy Coordinating Committee’s Action Plan.  Catherine Collin, Michael Lee, Jill Castle, Anessa Fehsenfeld, Chuck Riesbeck and Conrad Reynoldson provided various perspectives based on their family and individual experiences.  The topics the parents touched upon included newborn screen, variability of care and use of the CDC Care Considerations guidelines, problems with conducting clinical trials in the US such as the lengthy approval process, apparent duplication of certain studies and many studies being done overseas, not in the United States, cognitive issues and social challenges for families to include opportunities for adults seeking college and employment options.  The panel was moderated by Dr. Elizabeth McNeil from the NIH who asked questions of the participants to expand on their comments.  The floor was open to questions from the audience.  Among the topics discussed were issues related to available family resources, quality of life issues, college and post-college opportunities, hastening the clinical trials process while elevating urgency with regulators, rehabilitation programs and gaps in support for families as the boys get older.

 

More to come!

 

Brian Denger

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Comment by Brian Denger on February 14, 2011 at 3:33pm

Keith,

Elizabeth McNeil has left, but some things to bear in mind is the proposed cut is in the House version of the FY 2011 continuing resolution and the Senate version will probably be much less.  This differences will be worked out in committee.  Current programs, especially priorities will probably be left intact.  Areas that should be affected are pending or future grants and low priorities.  Recalling that much of the muscular dystrophy research at NIH resulted from the MD CARE Act, directed by Congress, I would be very surprised to see changes in these programs.  There is never a guarantee, but this seems to be a well supported opinion here.

 

Brian

Comment by Keith Van Houten on February 14, 2011 at 12:58pm
Brian - please ask Ms. Mcneil how much of the $1 billion cut in the NIH's budget that the GOP has proposed will impact research funding.  Do they expect a cut would come directly out of research expenditures, or some other area of their budget?
Comment by Leeandra Arhdeacon on February 14, 2011 at 12:33pm
Thank you for keeping us updated, we all wish we could be their with you.
Comment by Liisa Underwood on February 14, 2011 at 12:08pm
Wow!  Thank you for keeping us updated!  Big hugs to you all!
Comment by Connor's Dad - Brian on February 14, 2011 at 11:56am
Thanks for Sharing Brian - we really appreciate it.

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