It’s the official start of summer. And for our community, it’s that time of year when we come together with caregivers, scientists, and industry for PPMD’s Annual Connect Conference. This year’s agenda is quite astonishing, reflecting the vast number of therapies in the works, the updates to care, our increased presence in Congress, and a panel series that will dissect the clinical trial process from all points of view. We will also host a meeting prior to the Connect Conference, a roundtable meeting called Transforming Duchenne Care. We have gathered leadership from top medical institutions, neuromuscular experts, and patient representatives to discuss in an open forum the ideal configuration, features, services, and structure of state-of-the-art family centered care for patients with Duchenne. PPMD is proud of the research we continue to fund, and supporting potential therapies is one of our top priorities. But we also know the importance of improving care, educating the medical community, raising awareness, and maintaining federal support. Looking at this month’s newsletter, I’m proud of what this community continues to achieve, working together to end Duchenne. I’m also proud of one young woman who has made her voice heard in Washington the last several years: Elizabeth Heller. Elizabeth is the rarest of the rare – a woman with Duchenne. And she’s telling her story and using her voice so that people know that while Duchenne tends to predominantly affect men, woman can be affected. We are a community of fiercely passionate people and exceptions to any sort of norm. We are a community of people that will get what we want…we will end Duchenne.

 

I look forward to seeing many of you in Florida and hope the rest of you enjoy the official start of summer!

 

P.S. Many of you have emailed me, asking what I’m up to or if PPMD was represented at a certain meeting. Each month, I will try to bring you a brief recap of some of the places we’ve been and what we’ve been doing!

Where we are and what we’re doing

The end of April and May were particularly busy. June is shaping up to look the same!

The last several weeks:

  • 4/22: Conquer the Canyon
  • 4/27: Meeting with Drs. Katz, Unger, Temple, and Farkas to discuss clinical trials in Duchenne and specifically adaptive design. We discussed the importance of including boys across the spectrum of Duchenne and how trials might be adapted to be inclusive.
  • 4/30: Serve as Chairman of the DMDRP review committee. With sincere thanks to the PPMD advocacy community, Duchenne is included in the Department of Defense’ Congressionally directed research program. Round one of the competitive grants has been completed and grants awarded. We have expended the vision and guidelines for the next round which is expected to be announced in Fall.
  • 5/2: Symposium on Best Practices in clinical study design for rare diseases
  • 5/4: Met with Joe Clark, researcher at University of Cincinnati. His work is focused on autism and specifically Creatine Transport. We discussed the possibility of crossing the autism mouse model with Duchenne.
  • 5/9: Charlotte, NC – Inspired Wining (whining) to discuss women as primary caregivers and maintaining physical, emotional, and mental health.
  • 5/11: Transforming Duchenne Care. Developing the model for integrated care, challenges and opportunities. Steve Allen/CEO Nationwide Children’s and Elizabeth McNeil, MD will lead this discuss during the annual PPMD conference.
  • 5/14: NORD Corporate Council
  • 5/15-16: Presenter at the NIH/FDA meeting on the relevance of Natural History studies as a foundation for successful clinical trials.
  • 5/17-19: GoldLab Symposium. This involves academics, industry, and leaders in healthcare to discuss Health Economics and the impact of expensive therapies.
  • 5/21-22: Speaking at the ISCA consortium to address the need to include phenotypic information when reporting genetic testing results.
  • 6/6: Nationwide Children’s Hospital to discuss their clinical model.
  • 6/19: Speaking at BIO
  • 6/21: New Directions Stem Cell Workshop
  • 6/25: FACES Ohio Launch
  • 6/27-28: Transforming Duchenne Care (at Connect Conference)
  • 6/28-7/1: PPMD’s Annual Connect Conference

 

Pat Furlong, Founding President, CEO
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