This year’s  Annual Conference brings together more than 1,000 families, researchers, clinicians, and industry partners from around the world for three days of informative and interactive sessions featuring the latest news in research, clinical trials, approved and emerging therapies, care initiatives, quality of life issues, and more. This conference marks the largest in PPMD’s history! 

During her opening remarks on Thursday, June 29, PPMD Founding President and CEO Pat Furlong reflected on the past year, noting that this is an exciting and inspiring moment for the Duchenne community on the heels of the first ever gene therapy for Duchenne approved earlier this month, bringing the total  approved therapies for Duchenne to six, with hope that two pending therapies may also be approved within the year.

Furlong also highlighted an upcoming milestone for the organization, as 2024 will mark PPMD’s 30th anniversary. To celebrate, PPMD is offering a one-time-only special promotional ticket price of $30 for all family registrations for the 2024 Annual Conference, taking place June 27-30, 2024 in Orlando, Florida. Registration is now open and 2023 Conference attendees who register while on-site in Dallas, TX can receive a special gift that will help in getting ready for next year!

Register for 2024

Day 1 of this year’s Annual Conference kicked off with presentations and a Q&A session with Annemieke Aartsma-Rus, PhD, Craig McDonald, MD, and Kevin Flanigan, MD, who discussed current research, advancements in cell and gene therapy, and the importance of patient advocacy in shaping regulatory policies. Building on this, PPMD’s Niki Armstrong, MS, CGC and Lauren Stanford emphasized that there is no form of advocacy too small. For families and individuals wanting to make a difference for the Duchenne community, getting involved in advocacy, at the state and local level, and in the The Duchenne Registry, can be a great way to take action.

Advocate with PPMD The Duchenne Registry

ACCEPTING HELP

Both caregivers and individuals living with Duchenne and Becker heard from peers about the importance of accepting help. Parent and grandparent panelists emphasized that caregiver burnout is a very real concern, and offered advice on seeking assistance, whether it be from strangers, friends, or hiring professional caregivers. During the Teen and Adult Track sessions, PAAC member Adith Thummalapalli shared his experience going away to college and living independently, emphasizing that many people are willing to help but do not know how.

THURSDAY KEYNOTES

During his keynote presentation, PPMD Board of Directors member Chris Jones encouraged attendees to discover the magic of asking “what if?” Jones explained that asking “what if” can help families create something beautiful and bridge the gap between “I wish” and the often difficult reality of “what is.” 

When we gather at PPMD’s Annual Conference as a community, we are reminded just how important social connection is. Keynote speaker Brandon Kozar, PsyD, MBA noted social pain–exclusion, rejection, and breakups–are experienced in the same part of the brain as physical pain. Prioritizing healthy relationships is an essential aspect of caring for your mental health.

As so many families and individuals living with Duchenne and Becker know, a diagnosis may inform how you experience life, but it does not define who you are. Rachel Callander emphasized that words matter, and shared her Awesomeness Forms concept as a way for families to celebrate their children’s unique and wonderful qualities.

BECKER BREAKOUT

Dedicated programming for Becker muscular dystrophy included a breakout session exploring both clinical care and emerging therapies in Becker muscular dystrophy. Dr. Craig McDonald provided an overview of neuromuscular care, followed by cardiac care, genetics, and physical therapy considerations. We also heard from researchers in the space, including talks from Dr. Joanne Donovan on EDG-5506, Dr. Paula Clemens on the use of vamorolone in Becker, and Dr. Claudia Senesac on ImagingNMD and recent MRI findings and ongoing imaging studies in Becker. We look forward to continuing to dig in deeper and learn more about the natural history and needs of the Becker community!

TUNE IN VIRTUALLY

There’s plenty left in store for the next couple of days!

You can watch sessions from this year’s Annual Conference live by registering here. Both in-person and virtual attendees can access our Conference Hub website and app for an enhanced Conference experience.

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