On September 7, we will once again join forces with Duchenne and Becker organizations, families, partners, and all those like minded individuals around the globe to commemorate World Duchenne Awareness Day. This year we acknowledge and applaud the work being done to break barriers in the Duchenne community.
If you know me, you know that I am not a fan of barriers. I refuse to hear the word “no,” to sit back and accept the status quo. And I will continually fight to push the limits of our knowledge, our reach, and our ability to provide tangible help and hope for our community.
PPMD continues to expand our work, to break barriers so that all those living with Duchenne and Becker muscular dystrophy are looking to a brighter future. This year we saw the first gene therapy approval for Duchenne. The years of supporting research efforts to make this therapy a reality and countless hours of tireless work by clinic teams to bring ELEVIDYS to patients have finally seen their day.
In addition, where once traveling to just the one or two institutions known to understand Duchenne for care was the norm, now PPMD has a Certified Duchenne Care Center network of 38 institutions, spanning the country where families can go to receive optimal care. We have also begun the work of breaking barriers for our adults living with Duchenne and carriers also in search of better access to information and care specialists.
PPMD’s work in Washington, DC is instrumental in keeping the progress we see in the research field moving forward so that it isn’t just ideas, but rather treatments and therapies that bring real outcomes and real hope to those living with Duchenne and their families. Keeping a constant presence on Capitol Hill, at the FDA, and at other institutions reminds these policymakers that Duchenne and Becker are here, that our opportunities
matter, and that our community is willing to put in the work so that we can see a better outcome.
Breaking down barriers is not new to me or to PPMD. It is not new to our worldwide community. Fighting for our families, looking ahead at hurdles and thinking through how we can best move mountains, this is what breaking barriers is about. It is about the love of our community and the strength we have together. As Maya Angelou so beautifully said, “Love recognizes no barriers. It jumps hurdles, leaps fences, penetrates walls to arrive at its destination full of hope.”
Read PPMD’s 2023 World Duchenne Awareness Day / Duchenne Action Month press release here.