Sarepta’s 120 week stability data on both the six minute walk test, as well as, pulmonary function is good news for the Duchenne community. For the 12 young men in the study, we are thrilled. And for those waiting, we see hope on the horizon. We congratulate Sarepta for their continued commitment to people with Duchenne and we hope the FDA will consider the accelerated approval pathway.

PPMD’s Vice President of Research, Sharon Hesterlee, PhD, participated in a great piece published by Neurology Today on the potential impact of the FDA’s latest expedited review program — the breakthrough therapy designation — on the therapy pipeline for Duchenne, discussing both drisapersen and eteplirsen. Sharon makes it clear – this community will not accept the FDA’s lack of flexibility. We don’t have time.



This week, critical meetings are taking place in Washington, D.C. Our Director of Advocacy and Parent Outreach, Ryan Fischer, and I will be on Capitol Hill today meeting with Members of Congress regarding the MD-CARE Act (MDCA) reauthorization. Other advocates from the Duchenne community will be meeting with FDA, requesting the Agency to utilize existing vehicles such as Accelerated Approval to facilitate delivery of promising drugs to people who need and deserve the opportunity to stabilize progression, to protect and preserve muscle cells and muscle function. A Congressional Briefing will take place on Friday to inform and update representatives about progress to date and the urgency of getting treatments like eteplirsen to patients.

At the end of the month (and on the eve of Rare Disease Day), PPMD will return to D.C. for our Annual Advocacy Conference. While the MDCA will be the centerpiece of our agenda, the 2014 Advocacy Conference (Feb 23-25) will not just focus on this legislation. We plan to hold a meeting on the morning of Monday, February 24th to further our conversation from the recent Duchenne Policy Forum in December. As you are aware, the Duchenne community pledged to write draft guidance on Duchenne for the FDA, we’d like to update the community on the progress of that effort as well as re-engage with the community on the plan.

We will also hold a Congressional briefing on the MDCA on Tuesday February 25th, highlighting the progress made to date because of the bill, the impact of the Wellstone Centers of Excellence, and explain the current landscape where we have some real potential therapies getting closer to approval. We must continue to stress the urgency for getting therapies approved.

February continues to be the month that this community makes its mark on our nation’s capital. There are different Duchenne organizations with varying strategies using their voice in Washington throughout the month. And while we may differ in tactics, we are all after the same goal – to end Duchenne. When Rare Disease Day is recognized on February 28, we feel confident that our government will know what Duchenne is and why we must show flexibility and compassion NOW.

If you aren’t sure how you can help PPMD’s advocacy agenda or are unclear as to what our strategy is, please join us for next week’s webinar outlining what has been done and what we are doing…as well as how you can help.

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