This past weekend marked the third and fourth stops on PPMD’s Every Single [One] Tour – one taking place at Denver Children’s (University of Colorado) – one of PPMD’s Certified Duchenne Care Centers – and the other in conjunction with Lurie Children’s Hospital and Northwestern University in Chicago. The PPMD team was excited to divide and conquer for two great ESO events!
The event on Saturday in Colorado – with over 100 attendees, including families, presenters, and professionals – was a great example of why Denver Children’s is a PPMD Certified Duchenne Care Center. We had several members of the clinic team presenting and coordinating the day. Their dedication to the families they serve remains abundantly clear. We are grateful for the strong partnership we have with them and look forward to growing our collaboration over time.
We started the day all together and then split into two tracks: a family agenda and a professional agenda that offered continuing education credits, Addressing physical therapy needs in Duchenne presented by Terri O. Carry, PT, Children’s Hospital Colorado; Tina Duong, MPT, Lucile Packard Children’s Hospital Stanford; and Claudia Senesac, PT, PhD, PCS, University of Florida.
Recap of Presentations
Current Status of Drug Development and Clinical Trials
Abby Bronson began the day with an overview of clinical trials in Duchenne. She went over the various therapeutic targets, discussed which companies are currently enrolling patients in clinical trials, and what the study protocols entail (including number of patients, and inclusion/exclusion criteria). Information on clinical trials can be found on PPMD’s registry DuchenneConnect.org and clinicaltrials.gov. Abby’s talk segued perfectly into the next speaker, Dr. Julie Parsons.
Research at Children's Hospital Colorado
Dr. Julie Parsons gave a great overview about the muscle clinic team at Denver, many of whom have been with the hospital for over 10 years. She discussed the multi-disciplinary care model and the specialists involved in caring for Duchenne patients.
Dr. Parsons then gave an explanation about how the hospital chooses to run a clinical trial – how they make the decision about which studies to choose, the process involved to get them up and running, and how long it takes to start. Normally the process can take anywhere from 3-6 months. Typically their institution takes on trials entering into Phases 2 and 3. She then went over the studies being conducted currently at the clinic including Pfizer, Summit, Sarepta, PTC, Fibrogen, Italfarmaco, and Marathon.
DuchenneConnect & Decode Duchenne
Ann Martin, a genetic counselor and co-director of DuchenneConnect, discussed the importance of genetic testing and how the DC registry works to serve our families. She discussed the different mutations in Duchenne, as well as why it is important to know the mutation. Please be sure to check out www.DuchenneConnect.org for free genetic counseling or genetic testing through our Decode Duchenne initiative.
Christina Chan, MD spoke about the impact of Duchenne on the endocrine systems. She covered bone density, puberty, when a dexa scan is appropriate and what it means, testosterone treatment, the use of bisphosphonates, and treatment for osteoporosis.
Anne Stratton, MD went over Standards of Care for Adults and Children with DMD. Since the last update of the care considerations, there remains a gaps in care considerations for our community, particularly the adult population. Anne was involved in the process of updating the standards of care for Duchenne in children and adults. The MD-CARE Act Amendments mandated the updating of this document and Anne was part of the group of experts from around the world working on the update.
Stephanie Nakano, MD discussed caring for the heart. She went over the differences between echo and cardiac MRI and when they are needed. Stephanie also went over the various pharmacologic therapies used in Duchenne patients to include beta blockers, Angiotensin-converting enzyme (ACE) inhibitors, Angiotensin II receptor blockers (ARBs), and anti-coagulates.
The afternoon was filled with even more informative talks around caring for your child. Laura Watne discussed the importance of nutrition, as well as the need for monitoring and maintaining a healthy diet for all. Oren Kupfer discussed pulmonary care in patients and options for assistive breathing technology over the trajectory of the disease. Anne Stratton went over physical therapy, the dos and don'ts for patients, and what is important for stretching. Finally, Emily Maxwell gave a great overview of advocating for your child to ensure the most appropriate and inclusive education plan.
Our industry partners provided great overviews of their development programs. We’d like to thank Marathon, Pfizer, PTC, Santhera, and Sarepta for joining us and updating the community about their ongoing trials.
I finished the day with an update on our advocacy initiatives, providing a historical perspective on Duchenne advocacy and what efforts are ongoing and ahead for the community.
We’d like to thank Chris from Adaptive Adventures for giving the kids a great time on the hand bikes! The mission of Adaptive Adventures is to provide progressive outdoor sports opportunities to improve the quality of life for individuals with physical disabilities and their families.
One of PPMD’s well-known Run for Our Sons runners, Darrell Knight, also took time during the day to share his experience with the program. He runs for his son, Jack, and shared how he keeps himself motivated to continue to hit the road for various races across the country. Most poignantly, Darrell shared how he was able to train hard enough to provide Jack with a moment he’d wanted – the experience of a marathon finish line. After explain that he was able to carry Jack on his back the last tenth of a mile to the finish, Darrell challenged the entire audience to join the Run for Our Sons team at Rock ‘n Roll Denver on October 16. The series features a half marathon, 10K, & 5K. The team is growing and you can still get in on the action here: www.parentprojectmd.org/denver2016. Special thanks also to Colorado/Wyoming FACES Coordinator, Angela Knight for helping make the day a success!
I think everyone who participated in our Denver meeting had an incredible experience and we are excited about our stops later in the year! See the full schedule below and we hope to see you on the road!
About the Every Single [One] Tour
For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect Conference, FACES meetings, and Roundtables. The Connect Conference continues to grow with more researchers, clinicians, and pharmaceutical companies joining entire families who have found the conference as an opportunity to directly interact with thought leaders and reunite with friends.
In an effort to reach every single family facing a Duchenne diagnosis in the U.S., PPMD launched a multi-year community experience called the Every Single [One] Tour. Combining each of the pillars that make up PPMD’s mission – To End Duchenne – the Every Single [One] Tour brings updates on research, advocacy, and care to half a dozen cities in 2016.