The third and final day of PPMD’s 2023 Annual Conference kicked off with the much-anticipated Gene Therapy Town Hall. Buddy Cassidy opened the session by sharing his experience as a Patient Representative for the May 2023 FDA Cellular, Tissue, and Gene Therapies Advisory Committee meeting. Buddy’s remarks were followed by presentations from Solid Biosciences, REGENXBIO, Pfizer, Inc., and Sarepta Therapeutics, as well as Dr. Barry Byrne, Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. 

During the Town Hall, Dr. Chet Villa from the Heart Institute at Cincinnati Children’s Hospital discussed how we can move forward as a community with a therapy that offers a lot of promise but also has a lot of uncertainty, particularly when it comes to possible cardiac implications. Speaking during the “Ethics and Fair Play” session later in the afternoon on Saturday, Dr. Alison Bateman-House discussed ethics questions surrounding pediatric gene therapy trials.

TESTOSTERONE, PUBERTY, AND DUCHENNE

The “More Than Puberty: The Impact of Testosterone” session on Day 3 covered topics ranging from the role of testosterone and puberty in an individual’s health, the impacts and challenges of delayed puberty that is associated with Duchenne, and treatment options for addressing delayed puberty in Duchenne patients. Dr. David Weber explained that puberty is important for physical, cognitive, and emotional health, and urged parents and caregivers to meet with pediatric endocrinologists at an early age.

PAAC member Colin Werth shared his personal experience with testosterone therapy and offered parents and caregivers practical advice, touching on the positive impacts the treatment often has on self-esteem and quality of life. The Teens and Adults Track also met on Saturday to discuss self-esteem and challenges associated with family and privacy that individuals living with Duchenne often face as they get older.

AGE-SPECIFIC CARE

Attendees had the opportunity to deep dive into care topics specific to early childhood, adolescence, and adulthood during the “Care Corner” breakout sessions on Saturday afternoon. Each age-specific session featured brief overviews and Q&A panels on cardiac, pulmonary, and neurological care, as well as endocrinology, gastroenterology, and occupational and physical therapy.

Day 3 also featured time for 1:1 patient assistance programs and PPMD For You appointments, Kids and Sibs Track gatherings, and breakout sessions on a wide variety of topics, including searching for happiness and approaches to learning, school, and developing Individualized Education Plans (IEPs).

ADVICE FROM BEHAVIOR EXPERTS

Some individuals with dystrophinopathies like Duchenne will have cognitive, emotional, and social challenges that will require support. The “Battling Behavior” session on Day 3 explored neurodevelopmental disorders, psychiatric concerns, and behavior challenges often associated with Duchenne, with Dr. Natalie Truba sharing insights on where, when, and how to intervene if these issues begin to interfere with daily life. Education specialist Denise Gruender then provided practical tips for managing disruptive behaviors at home and at school.

BIG HOOPLA

After three full days packed with learning, networking, and engaging in sometimes difficult conversations, PPMD’s 2023 Annual Conference concluded with a Hoedown Hoopla. The celebration featured photo booths, games, face painting, and lots of dancing, and offered families a chance to connect, relax, and recharge with friends.

Thank you to our sponsors and all of the attendees for making this year’s Annual Conference both a fun and informative event! We are so grateful to be able to bring the Duchenne and Becker community together–we look forward to it every year!

CELEBRATE 30 YEARS OF PPMD

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!

Register for 2024