On March 4th I decided it was time to join PPMD on Capitol Hill and make my voice heard in the fight to end Duchenne. I was fortunate to have my dear friend Ann Dreher support me and join in the fight to urge members of the House and Senate to sign the 2019 Duchenne Muscular Dystrophy Appropriations Request letter. The appropriations letter, among other things, asks Congress to maintain level funding of $6 million for CDC's Muscular Dystrophy Program. Other requests included asking the CDC to support the advance of Duchenne newborn screening, requesting the NIH to demonstrate its stated commitment to improved research data-sharing, encouraging the FDA to continue implementing policies to promote access to information about how patient experiences data is used within reviews of newly approved products, requesting that the Social Security Administration make available data on the rate at which those with Duchenne utilize SSA programs, and seeking a report from Medicaid and Medicare on the use of the newly established ICD-10 code specific to Duchenne.
Ann and I were able to meet with both Senators from Ohio (Rob Portman and Sherrod Brown) and also our Congressman, Jim Renacci. During our meetings we educated them about Duchenne and gave them an overview of how Duchenne has affected our families. More specifically, I discussed the impact of Duchenne on RJ's daily life. In a nutshell, I told them our family has been navigating the Duchenne world for almost 10 years and it has completely altered our lives. Our family feels like we have been stuck in the eye of a hurricane and we can't get out. Our sweet 15 year old son RJ who is smart, funny, and hardworking stands strong no matter the daily obstacles in front of him. He is resilient but our family needs hope. We know time is running out and we need help to achieve advancements in research and hopefully one day a cure for Duchenne.
Another component of the Advocacy Conference included a one day Duchenne Patient-Focused Compass meeting. This meeting was taped and can be viewed below. In the audience were federal agency partners including members from the FDA, NIH, CDC, and CMS. It was a long, emotional day listening to panels of parents talk about the toll that Duchenne has taken on their sons and families, clinical trial and therapeutic experiences, and access to services and supports but the goal was to have the federal partners became more educated to the priorities of the Duchenne community.
I was so glad to have the opportunity to be an advocate with PPMD for the Duchenne Community. I hope all our efforts made a difference! Thank you again to Ann for taking up the fight to end Duchenne all the way to Capitol Hill!