Here's what the week looked like for me. On Monday I attended the Muscular Dystrophy Coordinating Committee meeting. As a reminder, this committee (MDCC) was created because of the MD CARE Act. The MDCC was charged with writing a comprehensive research plan for the muscular dystrophies. http://tinyurl.com/53g7sy
Presentations centered around investments in research, translation, clinical trials and burden of care. Understanding Burden, or the financial/emotional cost is essential in order to incentivize industry to develop therapies and to convince insurers that expensive therapies are cost effective when compared with disease burden. Amazingly, in a time when the NIH budget is very tight, the NIH has increased investments in DMD.
I am thankful to all of you for your help on the ReAuthorization of the MD CARE Act. We have crossed the 100 mark in terms of co-signers in the House and 22 Senators onboard. If the stars line up, this legislation will come out of Committee (HELP committee in the Senate and Energy and Commerce on the House side) and be sent to the Floor for vote during this Congress. If you haven't connected with your representatives, please do. If your representative has signed on, send them a note of thanks. Message can be really short/simple: It's Wednesday, thank you for your support of the Re authorization. It means so much to all of us.
Yesterday, I met with Carl Gist from Congressman John Barrow's office. Carl was wonderful and very supportive and hopefully Mr. Barrow will soon sign on. If you are from Georgia, please let them know you care about this. Also met with Sarah Bittleman from Congressman Darlene Hooley's office. Sarah was wonderful as well. If you live in Oregon, send them a note as well.
Today, I'm on my way to Philly. We have the virtual roundtable this afternoon.
Tomorrow, I'm meeting with a small committee to talk about 'google alerts', the laundry list of approved drugs what we might do as a community to accelerate our understanding, figure out if the evidence (mouse data) is sufficiently convincing or if the experiments need to be repeated in larger animals. It is so difficult to receive 'google alerts' every day about 'maybe something will have benefit' and not have a path to determine specifically 'yes' or 'no'.
Working to EndDuchenne for every boy/young man...