PPMD Community

PPMD’s #EverySingleOne Tour: Boston, MA

Boston made for a great location for our October Every Single One Tour stop. Nothing beats the northeast in the fall. Boston is home to a number of great Duchenne organizations, a network of active families, and where most of the drug companies involved in Duchenne are headquartered.

Thank you to everyone who volunteered, attended, and presented in Boston. It was a productive day of presentations and discussion about research, care, advocacy, community, and clinical trials.

Special thanks to JB’s Keys and all our industry partners for their sponsorship of this tour stop.

Active Clinical Trials & Research Overview

PPMD’s Founding President & CEO Pat Furlong started the day off with an overview of PPMD, Duchenne research, and a review of drug development strategies and current clinical trials (download).

Optimal Care in Duchenne

Regina Laine from Boston Children’s provided an overview of coordination of Duchenne care and explained what is available at Boston Children’s (download).
Dr. Jonathan Finder (Child Hosp of Pittsburgh) provided an overview of pulmonary care and management across the spectrum of Duchenne, including guidance on equipment (download). Dr. Finder is also a member of PPMD’s Board of Directors.
Traveling in from our nation’s capital, Dr. Chris Spurney with Children’s National, gave a talk on current cardiac care in Duchenne (download).
Molly Colvin, PhD gave a very informative talk about psychological issues that can be part of Duchenne and strategies to try to help with those issues (download).
Finally, Dr. Laura Case, addressed Physical Therapy needs and gave a hands on presentation that included equipment and a great demonstration from Jack Becker!

Genetics and You

PPMD’s own Ann Martin, co-Director of DuchenneConnect gave a talk on genetics and your family (download). Ann also emphasized the importance of DuchenneConnect, PPMD’s patient report registry. We encourage everyone to join this powerful registry. It is a simple and easy way for you to share your Duchenne experience and data to make a difference, from the comfort of your home.

Ann also gave information about PPMD’s Decode Duchenne Genetic testing program. Decode Duchenne provides free genetic testing for people with Duchenne and Becker. Finally, Ann discussed the important work PPMD is doing with Duchenne carriers as part of our carrier initiatives.

Run For Our Sons

Parent Chris Hancock gave an inspiring talk about participating in Run for Our Sons.

When he started running after his son's Duchenne diagnosis, he noticed that he was able to better focus on the challenges his family faced. "I had more energy and was stronger. Ask any parent in this room and they'll agree - we can all use as much energy and strength as we can get."

We're grateful to Chris for sharing his story about being part of the Run For Our Sons team at our recent Boston Every Single One tour stop, and we hope you will consider joining him at the upcoming Boston Run to Remember!

Local Resources

Parents Linda Hastings, Amanda Becker provided wonderful insights into local MA resources. Many in the room contributed to the discussion about how to navigate local systems. One of the key take aways from this presentation was to migrate these wonderful resources collected by parents in MA into the new PPMD Resource Center. We hope many others will help contribute information to grow this central hub of resources and information.

Advocacy

I provided an update on PPMD’s advocacy initiatives (download) encouraging everyone to get active with their house and senate members.

Industry Every Single One Tour Sponsors on Current Clinical Trials

Throughout the day the audience heard from our incredible list of industry partners developing potential therapies for Duchenne.

Participants included:

Capricor – Leland Gershell, MD, PhD (download)
Catabasis – Joanne Donovan, MD (download)
PTC – Allie Freitas (download)
Pfizer/Bamboo – Sharon Marraffino (download)
Roche – Michelle Krishnan, PhD (download)
Santhera – John Karafilidis, PharmaD (download)
Sarepta – Siobhan Fitzgerald
Summit – Michelle Avery (download)

Thank you ALL for informing our families about your current trials. We all appreciated you being there!

About PPMD's Every Single One Tour

Combining each of the pillars that make up PPMD’s mission, the Every Single One Tour brings updates on research, advocacy, and care to families across the country, featuring a roster of leading experts in the Duchenne space. This is also an opportunity to connect with local families and, when possible, explore your area Certified Duchenne Care Center. As always, each meeting is free with breakfast and lunch provided. Kids are also welcome to attend and participate in PPMD’s Kids Track.

Upcoming 2017 stops: