If you are here at PPMD’s Annual Conference this year, you’ve likely seen a man with a camera running around snapping pictures. Rick Guidotti, photographer and Founder & CEO of Positive Exposure, delivered keynote remarks on Friday, June 30, sharing his work of photographing rare disease and changing how the world sees difference. Giudotti is on a mission to create space to celebrate diversity, and he will be photographing the Duchenne community as we gear up for PPMD’s 30th anniversary in 2024.

MENTAL HEALTH

You do not need to be in crisis to need and seek help for your mental health, emphasized panelists during the “Mental Wellness: Finding Balance” session on Friday. Mothers Jess Divin and Jill Castle urged families to focus on the present, noting that there is a significant difference between existing with Duchenne and living with Duchenne. For parents and caregivers, prioritizing mental health is essential to being the best possible support system and example for your child.

RESEARCH ROW

The “Research Row” sessions on Day 2 gave attendees an opportunity to hear directly from industry partners about the research they’re doing, their drug development process, and the therapies they currently have in the pipeline or on the market. Each company provided updates on the status of where their treatment is in the approval pipeline (if not already approved), spoke to the the benefits and side effects of their treatment, and answered questions from the audience on everything from expected label to dosing frequency.

Among the updates shared was Capricor’s announcement of positive 24-month results from the ongoing HOPE-2 open label extension study of CAP-1002. Read more in Capricor’s press release here.

PPMD is committed to enhancing the drug development process to ensure we have an environment that increases the likelihood of approvals and the speed of development. The Duchenne Drug Development Roundtable (DDDR), composed of industry partners, convened for dinner to address drug development challenges in a pre-competitive space.

CARDIAC CARE

During the “Stronger with Every Beat” session, clinician panelists discussed the typical progress of heart function for individuals living with Duchenne, offering insights on what families may be able to expect as their child grows. Drs. Chet Villa and Diepanjan Nandi shared information about cardiac management with medication and devices. Dr. Ryan Butts discussed the transition from pediatric to adult cardiac care for Duchenne patients, emphasizing that while the transition can be challenging, it ultimately means an increased life expectancy.

GI & GU CARE

When it comes to addressing gastrointestinal, genitourinary, and swallowing challenges, Dr. Richard Shell emphasized that therapies for Duchenne patients are focused on quality of life. Clinicians during the “Ins and Outs” session stressed the importance of hydration and explained how these aspects of an individual’s health are interconnected.

SOMETHING FOR EVERY MEMBER OF THE FAMILY

One of our favorite aspects of the Annual Conference is the unique opportunity for families to spend time with peers, whether that be fellow parents, carriers, grandparents, siblings, or individuals living with Duchenne. Day 2 of this year’s Conference kicked off with breakfast breakout sessions for family members to connect. Specialty tracks throughout the day also offered kids the change to engage and have some fun together. Once Conference sessions concluded for the day, families took an exciting excursion to the Dallas Aquarium for some interactive quality time.

TWEENS, TEENS & ADULTS

The Tweens and Teens & Adult sessions on Day 2 were filled with engaging conversations about everything from hobbies, to school, to favorite parts of Annual Conference. PAAC members gave advice to tweens about responding to challenges, with Jack Ridolphi sharing his experience with therapy and the benefits of speaking with someone about your thoughts, feelings, challenges, successes, and everything in between. PAAC members also spoke about self-esteem and social isolation, exchanging helpful tips and offering a safe space for young people to connect with peers.

TUNE IN VIRTUALLY

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.

CELEBRATE 30 YEARS WITH 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 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