PPMD recently hosted our fifth in-person Duchenne Healthcare Professionals Summit in Sanibel, FL. Over the course of three impactful days, we were joined by around 300 healthcare professionals, researchers, and industry partners to collaborate, connect, and share knowledge, with the goal of learning from one another to ensure that caring for individuals with the latest information and technology remains a constant priority. The dedication to people living with Duchenne and Becker was evident among all attendees.
Recognizing Progress & Looking Ahead to the Future
PPMD’s Vice President of Clinical Care & Education, Rachel Schrader MS, APRN, CPNP-PC, kicked off the 2024 Summit addressing PPMD’s 30th year anniversary and our commitment to fighting for every future. Rachel spoke about the progress that has been made since PPMD’s President & CEO, Pat Furlong founded the organization in 1994, and our continued priority to advance care and improve access to treatments for every person affected by Duchenne and Becker.
Carrying forward this theme, keynote speaker Dr. Jerry Mendell spoke about his 40+ year career, from his education and training, to experiencing the implementation of steroids, exon skipping therapy, and development of gene therapy. Dr. Mendell received a standing ovation following his talk for his commitment to patients living with Duchenne.
Fostering Discussions to Advance Care & Treatment
50 neuromuscular centers from across the country were represented at this year’s Summit. This allowed for invaluable connection and collaboration among specialists with similar interests and goals. Dr. Cuixia Tian from Cincinnati Children’s Hospital Medical Center, one of PPMD’s Certified Duchenne Care Centers, emphasized the value of learning from peers, citing insights gained as instrumental in enhancing care for Duchenne patients and families:
“My favorite aspect of the Summit is all the valuable opportunities to learn from our peers in the field throughout the Summit, from [expert presentations] and casual networking lunch conversations, to organized discussion during the directors meetings, and more. The information we learned from the Summit will help us to improve our care for our Duchenne patients and families. Learning from colleagues’ research experiences helps us to be more prepared for upcoming new medication approvals.”
– Cuixia Tian, MD, Cincinnati Children’s Hospital Medical Center
PPMD’s Summit also serves as a unique forum for those in the field to dive into critical issues and gaps in care, and begin to work towards finding solutions. Many barriers to reaching these goals require ongoing partnerships with government agencies to accelerate regulatory processes and improve access to new therapies. We are grateful to Peter Marks, MD, PhD, Director of the Center for Biologics Evaluation and Research (CBER) at the U.S. Food and Drug Administration (FDA), who traveled to Florida to discuss challenges seen in rare diseases:
“Attending the PPMD Summit provided an opportunity to interact with a diverse group of individuals who all deeply care about making a difference as soon as possible in the lives of those with neuromuscular disorders. Being able to interact with patients, parents, nurses, allied professionals, and physician experts all in one place is truly motivating.”
– Peter Marks, MD, PhD, Director, CBER, FDA
Join us at PPMD’s 30th
More of the learnings and topics discussed at the Summit will be shared at PPMD’s 30th Annual Conference this summer in Orlando, Florida – register today to join us June 27-30, 2024! Registration for virtual participation will also be available soon.