PPMD was thrilled to head south to Dallas Texas for our October End Duchenne Tour stop on October 13, 2018. More than 80 people came together to learn about the latest information and resources for Duchenne families. PPMD was grateful to a number of the UT Southwestern team for spending their Saturday with us to connect with local families.
Join the Fight
Join the Race to End Duchenne team in March at the 5th annual Skyline Half Marathon, 5K & 10k! Runners will travel along the Trinity Skyline Trail, the newest trail in downtown Dallas. Participants will run under the Margaret Hunt Hill Bridge and close to the Trinity River, all while maintaining gorgeous views of the Dallas skyline. For more information or to register visit the Race to End Duchenne website or contact Nicole Herring at firstname.lastname@example.org.
The Decode Duchenne program, offering free genetic testing and counseling for people with Duchenne, is now available for carriers – learn more!
- Speaker: Susan Iannacone, MD, UT Southwestern Medical Center
- Overview: Standard of care is still steroids and Susan discussed different dosing regimens being used by clinicians. Susan went through the current research strategies in Duchenne, the companies conducting trials, and the details associated with each trial.
- Take home: There are a number of exciting strategies currently underway in Duchenne. Talk with your care provider about opportunities in clinical research, and be sure to register with PPMD’s Duchenne Registry to hear about recruiting trials.
- (Download presentation slides)
- Speaker: Rhonda Bassel-Duby, PhD, UT Southwestern Medical Center
- Overview: Rhonda is from UT Southwestern Medical Centerand provided an update on gene editing/CRISPR Cas9 therapeutic strategy. CRISPR/Cas9 was adapted from a naturally occurring genome editing system in bacteria. The bacteria capture snippets of DNA from invading viruses and use them to create DNA segments known as CRISPR arrays. The CRISPR arrays allow the bacteria to “remember” the viruses (or closely related ones). The bacteria then use Cas9 or a similar enzyme to cut the DNA apart, which disables the virus. This could potentially be an ideal strategy for Duchenne.
- Take home: This is still in early stages, it has been completed in a a dish, in mice, and now in the dog model. The hope is to see this get to the clinic in the next 3-5 years. UT Southwestern Medical Center is a Wellstone Center funded by the government as a results of the MD-Care Act, PPMD-driven legislation in 2001.
- (Download presentation slides)
- Capricor (Download presentation slides)
- Catabasis (Download presentation slides)
- Santhera (Download presentation slides)
Dr. Pradeep Mammen, UT Southwestern Medical Center, spoke about cardiac issues in both patients and Duchenne carriers. He discussed current strategies for monitoring and treatment. (Download presentation slides)
Take home: All Duchenne patients require a full cardiac and pulmonary assessment. Cardiovascular complications in Duchenne patients and Duchenne carriers are common. Cardiac MRI is the preferred imaging tool to accurately assess the cardiac structure of the Duchenne patients.
Dr. James Poysky, Katy Child Psychology Associates, spoke about Duchenne and the brain. (Download presentation slides) Including:
- Introduction and causative factors
- Neurobehavioral Disorders in Duchenne
- Coping and Emotional Adjustment
Take home: There is an increased risk in Duchenne for issues like ADHD, OCD, and depression. These issues are often overlooked and should be addressed with your doctor. You can learn more about learning and behavior issues in Duchenne by visiting our website.
Leslie Nelson, PT, PhD, UT Southwestern Medical Center, spoke about PT management for Duchenne patients. (Download presentation slides) Including:
- What we know about exercise
- PT Management across the spectrum (Stretching/Splinting/Positioning)
- Clinical trials
Take home: Stretching is important at all stages of disease. A number of resources exist on the PPMD website and are available for both families and PTs.
PPMD & Community Speakers
- Ryan Fischer, PPMD, provided an overview of PPMD and a focus on the impact of Duchenne Advocacy on therapy development. Be sure to save the date for the 2019 Advocacy Conference! (Download presentation slides)
- Jen Ely, CGC, PPMD, spoke about Decode Duchenne and the importance of The Duchenne Registry. If you have not registered, please do. If you have registered, please update your information! (Download presentation slides)
- Aimy Aikens, Duchenne Family Assistance Fund, spoke about the programs administered by Little Hercules and Team Joseph to help families navigate insurance and provide equipment and support to families around the country. (Download presentation slides)
Race to End Duchenne
PPMD Board Chairman and longtime Race to End Duchenne teammember John Killian, shared his experiences going the distance to end Duchenne and encouraged local runners (and runners to be) to join him at the 5th annual Skyline Half Marathon, 5K & 10k! Runners will travel along the Trinity Skyline Trail, the newest trail in downtown Dallas. Participants will run under the Margaret Hunt Hill Bridge and close to the Trinity River, all while maintaining gorgeous views of the Dallas skyline. For more information or to register visit the Race to End Duchenne website.
Thank you to our industry partners for helping make today possible. A huge thank you to a number of the UT Southwestern team for spending their Saturday with us to connect with local families.
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.
Remaining 2018 Schedule:
Upcoming 2019 Schedule*:
- Las Vegas, Nevada – Winter 2019
- Omaha, Nebraska- Spring 2019
- Portland, Maine – Spring 2019
- Wilmington, Delaware – Fall 2019
- Memphis, Tennessee – Fall 2019
*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.