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!