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PPMD’s #EverySingleOne Tour: Iowa City, IA

Iowa Children’s Hospital marked our third stop on PPMD’s 2017 Every Single One Tour, held on April 1, 2017. The amazing staff at Iowa Children’s Hospital helped make this a wonderful day and we can see why they are one of the 15 PPMD Certified Duchenne Care Centers.

The care team, lead by Dr. Kathy Mathews, are an amazing group of providers. The families were engaged and appreciative of the information provided by the team and the other presenters. We are thankful to the team at Iowa Children’s for their commitment to superb care for the Duchenne Community.

 

Recap of Presentations

 

Active Clinical Trials & Research Overview

 

Dr. Mathews gave an overview of the basics of Duchenne stressing that Duchenne is one of over 40 neuromuscular diseases. She explained the cause of Duchenne and how there is a continuum between Duchenne and Becker. She then shifted her talk to focus on research by first outlining the various ways we try to understand a disease better such as having good natural history studies and registries such as DuchenneConnect. Iowa is one of the 6 sites in the country for MD STARnet which is aimed at comprehensive reviews of medical records to learn more about Duchenne.

She then turned her focus to research around potential treatments and outlined the phases of human trials and what each stage involves. Dr. Matthews then reviewed the clinical trials available in the area to patients and families. She concluded with a description of how the cancer treatment model has evolved over time. Years ago, if a child was diagnosed with childhood leukemia, >90% of the time it was fatal. Now most children are cured. This treatment model changed over time with continuing trials, careful matching of patients to treatments, combination therapies and aggressive management of complications. This will need to happen in the Duchenne community as well.  

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Optimal Care in Duchenne

 

PPMD’s Kathi Kinnett provided an overview of what comprehensive care is and what it means for a clinic to be a PPMD Certified Duchenne Care Center (CDCC). She stressed the importance of this not only for providing optimal care for patients but also making sure that our clinical trials are occurring in centers where optimal care is being provided so that any outcomes can be comparable. The CDCC program also relies on input from families so we encourage each family to take the Clinical Experiences survey, which tells the committee what type of care they are receiving and if there are issues with care, what those needs are. The Care Guidelines that the CDCC’s follow are currently being updated and will be published in the near future.

We then had the chance to hear from several clinicians that practice at Iowa Children’s Hospital about different parts of the care model.

 

  • Erik Edens, MD – gave an overview of Duchenne and cardiac care stressing that cardiac care should begin at the time of diagnosis and include a baseline echocardiogram and ECG
  • Stephanie Borst, RD – reviewed why it is important to have a good diet with the diagnosis of Duchenne and gave some good tips to help manage this: avoid sugary drinks, make sure you have a colorful plate (and M&M’s don’t count), shop the perimeter of the store for healthier choices such veggies, fruit, meat, fish, dairy, and avoid processed foods
  • Jose Morcuende, MD – gave an overview of bone health with considerations about contractures, fractures and biopsies as well as spinal management
  • Seth Perlman, MD – reviewed Corticosteroids and Duchenne. He echoed previous statements from Kathi Kinnett around the being careful during times of stress and what to do during that.
  • Tim Starner, MD – gave an overview of concerns around breathing and airway clearance in Duchenne and declared oral secretions public enemy #1 of those with Duchenne. He discussed ways to help clear secretions and ways to help with breathing
  • Christina Trout, MSN discussed her role as a clinical coordinator and nurse and ways she can help families make sure they are receiving the services and care that is recommended
  • Jim Porter, LISW – explored chronic sorrow and coping strategies to get through the tough times
  • Katie Laubscher, PT, DPT, PCS reviewed how people with Duchenne need to get activity they also need to balance this so that they are not damaging their muscles. She encouraged activities that can provide social interaction, are fun and promote self esteem
  • Shelly Mockler, PT, DSc, PCS – reviewed some of the various choices available to families with mobility aids such as scooters and wheelchairs and things to think about when considering them

 

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Genetics, DuchenneConnect and Your Family

PPMD’s Ann Lucas, co-Director of DuchenneConnect gave a brief overview of the DuchenneConnect Registry, PPMD’s patient report registry. This is a powerful tool for the community. DuchenneConnect has close to 4,000 registrants and would love to have everyone with  Duchenne, Becker and women who are carriers be a part of this Registry. Data from the Registry helps inform researchers as they plan clinical trials and research studies.  She reviewed the importance of knowing the exact genetic mutation for each family and that one of the programs PPMD has to help support this is our FREE genetic testing program, Decode Duchenne. Please be sure to check out DuchenneConnect.org for more information and don’t forget to join the Registry if you haven’t already!

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Industry Every Single One Tour Sponsors on Current Clinical Trials

Throughout the day the audience heard from our incredible list of companies developing potential therapies for Duchenne. Thank you all for informing our families about your current trials. They so appreciated you being there!

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PPMD, Advocacy, and You

PPMD’s Danielle Garrigan discussed the various ways families can get involved in the grassroots efforts of PPMD. Programs such as the End Duchenne .1K, Run For Our Sons, and Coach To Cure MD. She also discussed some of the exciting things coming up for our Duchenne Action Month this September to coordinate with World Duchenne Awareness Day on September 7th.  

We also enjoyed hearing from one of the families about their experience over the last two years participating in Coach to Cure MD. Sounds like the whole family enjoyed the time and it was an easy thing for them to do. The date for Coach to Cure MD this year is September 30th. We are always looking for families to participate – contact danielle@parentprojectmd.org for more information. 

The day wrapped up with Annie Kennedy providing an update on PPMD’s Advocacy initiatives, giving a historical perspective on Duchenne advocacy and what efforts are ongoing and ahead for the community. She also updated the group on our efforts in Benefit Risk, 21st Century Cures, and ongoing federal policy initiatives giving them a call to action to get involved!

Download Presentation Files: 

Links for Getting Involved:

 

About PPMD's Every Single [One] 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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