September 24, 2019 / Community

End Duchenne Tour Recap: Wilmington, Delaware

Parent Project Muscular Dystrophy by: Parent Project Muscular Dystrophy

PPMD’s End Duchenne Tour made a stop in Wilmington, Delaware on September 14. We were joined by over 100 people, including 30 families, 20 healthcare professionals and researchers, 15 industry partners, and 5 members of the neuromuscular clinic team from Nemours/Alfred I. duPont Hospital for Children.

We want to thank families for traveling from Maryland, New Jersey, Virginia, Pennsylvania, New York, and Delaware to spend the day with us!

Join the Fight

Stay involved and continue to meet other Duchenne and Becker families throughout the year. You can do in several ways, including joining our Race to End Duchenne team at one of these upcoming endurance events.

Join the Race to End Duchenne Team

David Hofstein, grandparent and PPMD board member, spoke about his experience participating in the 2020 TD Five Boro Bike Tour with the Race to End Duchenne team, which will be taking place again May 3.

David also spoke about joining the Race to End Duchenne Team for the upcoming 2020 Rock ‘N’ Roll Washington DC Marathon on March 28. Runners can choose from a 5K, half marathon, or full marathon. For more information, please contact nicole@parentprojectmd.org.

PPMD’s Connect

You can also stay in touch with the community by joining PPMD’s Connect, the official volunteer, parent-lead outreach program of PPMD. PPMD’s Connect groups plan social gatherings and serve as sources of information and support for local families. There are a few groups in the local area, and we encourage you to reach out via email or join a closed social media group made up of other Duchenne and Becker families.

PPMD’s Connect New Jersey

PPMD’s Connect Western Pennsylvania

PPMD’s Connect Virginia

PPMD’s Connect DMV (DC, Maryland, Northern VA)

Decode Duchenne

The Decode Duchenne program offers free genetic testing and counseling for people living with Duchenne, is now available for carriers – learn more!

Care Highlights

Orthopedic Standards of Care

Will Mackenzie, MD

  • Dr. Mackenzie presented on the screening and management of orthopedic issues in Duchenne – with the use of steroids, thankfully the need for scoliosis surgery has been drastically reduced.
  • Managing bone health is very important in preventing injuries; he emphasized having adequate levels of calcium and vitamin D help to keep bones healthy.
  • Annual screening for bone mineralization with DEXA and vertebral compression fractures with lateral lumbar x-rays enables appropriate management with bisphosphonates.
  • Stretching and wearing AFOs from an early age help to prevent contractures.
  • Download presentation slides

Physical Therapy

Laura Dougherty, PT, DPT, PCS

  • Laura gave a comprehensive physical therapy overview focusing on long-term goals in Duchenne to include maintaining function and comfort while facilitating independence.
  • Laura also discussed the benefits of safe exercise and activities with peers and suggested swimming and/or aquatic therapy.
  • Laura also met with families throughout the day to provide individual guidance and resources.
  • Download presentation slides

Psychosocial Standards of Care

Stephanie Chopko, PhD

  • Dr. Chopko presentation gave an overview about potential psychosocial impacts of Duchenne and how parents and healthcare providers can monitor, test, and intervene early.
  • Recommendations include community and social training, peer support, routines, consistency, and communicating among all members of your child’s healthcare and school teams.
  • Download presentation slides

Neuromuscular Standards of Care

Mena Scavina, MD

  • Dr. Scavina explained that although Duchenne follows a predictable path, every person is different and it is important to see a neuromuscular specialist (NMS) every 6 months to ensure your child stays as active and independent as possible.
  • Dr. Scavina closed with a list of colleges her patients have attended independently.
  • Download presentation slides

Research Updates

Duchenne Research Overview: The Landscape and Opportunities

Pat Furlong, PPMD

  • Pat gave an introduction to clinical trials in Duchenne including study types and the importance of being informed about a particular trial before participating.
  • Pat also gave an overview about the different types of drugs currently being investigated in the current robust Duchenne pipeline.
  • Download presentation slides

How to Advocate for Your Child with DMD “Learning the Lingo”

Michael Lo Sapio, parent

  • Michael, a father of two sons with Duchenne, acts as a clinical trials liaison for Nemours Children’s Hospital, and assists other families in navigating the regional clinical trial network.
  • Download presentation slides

Cardiovascular Consequences of Duchenne and Becker

Melissa Witman, PhD, University of Delaware

  • Dr. Witman spoke about an ongoing research study about the heart and associated blood vessels throughout the body and how they change over time in Duchenne and Becker.
  • This study uses ultrasound, echocardiogram, and cardiac MRI to assess the heart and blood vessels in children and adults living with Duchenne and Becker ages 8-21.
  • Download presentation slides

Industry Updates

 

PPMD & Community

Living with Duchenne

Jordan Reidenberg

  • Jordan, age 17, asked to speak to the attendees about his experience living with Duchenne.
  • Jordan is an social, independent teen who enjoys teaching others about Duchenne and sharing tips and tricks he has learned to modify his daily life.

About PPMD

Kathi Kinnett, MSN, APRN (PPMD)

  • Kathi spoke about PPMD’s core tenants, including PPMD’s efforts in Care (including the Certified Duchenne Care Centers), Advocacy, Research, and Community Engagement.
  • Kathi shared many ways to get involved, including the PPMD’s 2020 Advocacy Conference March 1-3 in Washington, DC to help us raise our voices on Capitol Hill!
  • Download presentation slides

Genetics, Your Family, and The Duchenne Registry

Jen Ely, CGC (PPMD)

  • Jen gave an overview of genetics in Duchenne and explained the different types of mutation in the dystrophin gene and what they mean.
  • Jen also spoke about Decode Duchenne and the importance of The Duchenne Registry. Although the registry is undergoing a transition to an app-based platform that we are excited to launch in the coming weeks.
  • Download presentation slides

Thank You!

A huge thank you to the Nemours/Alfred I. duPont Hospital for Children’s neuromuscular team, especially Dr. Scavina and Jen Hultberg, for hosting this event!

We also want to thank Michael Lo Sapio and his students from Salesianum School for volunteering in the Kid’s Room!

Next Up

In an effort to reach every single family facing a Duchenne diagnosis in the U.S., PPMD’s End Duchenne Tour brings updates on research, advocacy, and care to cities across the country, featuring a roster of leading experts in the Duchenne space.

You have told us what topics are the most important to you and we have listened, using your feedback to inform our robust agenda at each tour stop. 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 2019 Schedule

Upcoming 2020 Schedule*

End Duchenne Tour

  • Atlanta, Georgia – Winter 2020
  • Milwaukee, Wisconsin – Spring 2020
  • St. Louis, Missouri – Spring 2020
  • Portland, Oregon – Fall 2020
  • Long Island, New York – Fall 2020

End Duchenne Tour: Connect Sessions

  • Houston, Texas – Winter 2020
  • Miami, FL – Fall 2020

*Registration typically opens 1-2 months prior to each event. Visit EndDuchenne.org/Tour for more details and make sure you are signed up to receive emails from PPMD to be notified when registration opens.

Join Our Mailing List