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PPMD’s #EverySingleOne Tour: Los Angeles, CA

Children’s Hospital Los Angeles (CHLA) marked our first stop of the New Year on PPMD’s 2017 Every Single One Tour, held on January 21, 2017. We were thrilled to not only be holding another tour stop at one of PPMD’s Certified Duchenne Care Centers (CDCC) but also to welcome representatives from another CDCC – the neighboring Center for Duchenne Muscular Dystrophy at UCLA.

Throughout the day it was apparent that the team at CHLA led by Dr. Leigh Ramos-Platt, provides a wonderful caring experience for patients and families and that they have a good working relationship with the team at the UCLA CDCC.  We are so grateful to both these teams for taking care of the Duchene community in Southern California.

 

Recap of Presentations

 

Active Clinical Trials & Research Overview

 

Our first presenter, Dr. Stan Nelson (download presentation), gave an overview of the drug development process and strategies in the Duchenne community.  He then took the opportunity to go into more detail about specific clinical trials available in the area to families.  He discussed that a new undergraduate course has been developed as an educational tool around research using Duchenne as a model disease.  This course should be available online in the Fall 2017. He also discussed the importance of resources such as the DuchenneConnect patient registry and the value that this data can provide to the community.

This was a good segue way to the next presenter, Ann Lucas (download), co-Director of DuchenneConnect. 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.

 

The highlight of the morning was hearing from Dylan Miceli-Nelson, a young man with Duchenne (and Drs. Stan Nelson and Carrie Miceli’s son) who has participated in clinical research. He gave an overview of what the process has been like for him as a young teen in high school and some of the things every family should consider when thinking about participating in research. He said the key to making any research study successful was to provide plenty of snacks!  We couldn’t agree more!

 

Optimal Care in Duchenne

 

After PPMD’s SVP of Clinical Care, Kathi Kinnett (download), provided an overview of what comprehensive care is and what it means for a clinic to be a “PPMD Certified Duchenne Care Center,” we heard from several clinicians that practice at CHLA about different parts of the care model.

 

  • Leigh Ramos-Platt, MD and Arlene Deseo – along with other team members gave an overview of what the clinic visit looks like at CHLA and how they are dedicated to making sure each and every person gets the care they need.
  • Ajay Kasai,MD (download) – gave critical information on providing pulmonary care management
  • Jennifer Su, MD (download) – gave an overview of Duchenne and cardiac care
  • Lindsay Andras,  MD (download) – gave an overview of bone health with considerations about spinal issues

 

Run For Our Sons

Beth Kilgore (download), a runner and Duchenne mom, gave a motivating talk about how she has been a part of the Run for Our Sons program and how everyone can get involved. Thank you Beth for everything you do! This program is vital to the success of PPMD and the many different areas of research, advocacy, and care that we lead. We couldn’t do it without the support of each person in this program.

 

Advocacy

Ryan Fisher (download) provided an update on PPMD’s advocacy initiatives, giving a historical perspective on Duchenne advocacy and what efforts are ongoing and ahead for the community. He also updated the group on our efforts in Benefit Risk, 21st Century Cures, and ongoing federal policy initiatives giving families a call to action to get involved!

 

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 (download) – Houman Hemmati MD, PhD
  • Marathon (download) – Pete Sazani, PhD
  • PTC – Allie Freitas
  • Pfizer (download) – Rodger Kobes, MD
  • Santhera (download) – Jodi Wolff, PhD
  • Sarepta – Lisa Borland

 

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

Psychosocial issues in Duchenne

Dr. James Poysky (download) gave a very informative talk about psychological issues that can be part of Duchenne and strategies to try to help with those issues.  For more written information about these issues, please see the PPMD Learning and Behavior area.

 

Physical & Occupational Therapy

The day finished with a presentations about Addressing Duchenne Lifelong Therapy Needs and a great hands on presentation that included equipment and show & tell led by Leslie Vogel, MSPT (download).

 

About the ESO Tour

For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect ConferenceFACES 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 cities across the country.

Upcoming stops:  

View PPMD's 2017 Every Single [One] Tour schedule & find a stop near you!

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