As we say goodbye to September and hello to autumn, I wanted to take a moment and thank you.
PPMD and the entire Duchenne community are busier than ever. There is so much to do, to push forward, to get done, in our daily lives and in the care of our sons. Every now and then, though, it is important to take a minute to exhale and realize what you’ve done, what’s been accomplished, and what is happening around you.
I think it is safe to say that more was happening in September in this community than perhaps at any other time. As the kids headed back to school, we all rolled up our sleeves and we got to work as a community, as a team, as One Voice. In September, alone:
- We awarded three cardiac-specific exploratory awards for $50,000 each to Ohio State University and Nationwide Children’s Hospital.
- We awarded a $98,000 End Duchenne Grant to University of Pennsylvania to continue utrophin upregulation research.
- Prosensa announced the advancement of three exon skipping compounds.
- We joined the latest presentation in our DuchenneConnect Direct Access Webinar Series, this time from Dr. Kathryn Wagner regarding transition care for older patients.
- The FDA proposed an agreement to address the unique challenges associated with developing therapies for Duchenne and other rare diseases: PDUFA Reauthorization Performance Goals and Procedures, Fiscal Years 2013 Through 2017.
- Grassroots events were held around the country, raising tens of thousands of dollars for research thanks to: 3rd Annual Labor Day Classic; Brunch & Munch to End Duchenne; Comic Relief 2; Miles for Matthew Golf Outing and Miles for Matthew 5K/10K; Cassidy Family Swing for a Cure; Team Saij - Simply the Best; and Tee Up for Tanner.
- The 4th Annual Coach To Cure MD happened September 24 including the participation of over 500 universities and high schools, 10,000 coaches, and millions of fans. With donations continuing to come in via online and text, we will total over $1 million since the program began in 2008.
- Run For Our Sons welcomed a new race – Rock ‘n’ Roll Philadelphia ½ Half Marathon – and we celebrated our largest, most successful team at one of our first races – Disneyland Half Marathon. I couldn’t have been happier joining this year’s amazing team myself over Labor Day Weekend!
- WebMD featured Duchenne in its section on chronic disease: A Different Normal.
There have been entire years where we didn’t have this much happening in the community. And here we are, the first month of the school year with so much to be thankful for. October is looking equally, if not more, promising. We have great events lined up from Rally for Ryan, Rosstoberfest, and Sam’s Day; Run For Our Sons takes over marathons in Chicago, Denver, Hartford, & St. Louis; our DuchenneConnect Webinar Series continues with a focus on genetic testing; and we will continue to fund therapies and update care resources.
Time often feels like our biggest enemy in the fight to end Duchenne. But we can beat the clock if we stay in the fight, continue to collaborate, and support each other when we’re down. Together, we will end Duchenne.
Thank you for all that you do to support everyone living with Duchenne.