PPMD and NHLBI Co-Sponsor Duchenne Cardiac Working Group

Parent Project Muscular Dystrophy (PPMD) has partnered with the National Heart, Lung, and Blood Institute (NHLBI) to convene a working group titled “Contemporary Cardiac Issues in Duchenne Muscular Dystrophy.” The group, made up of top pediatric and adult cardiologists as well as clinical researchers, will meet July 10-11, 2014 in Bethesda, Maryland to address critical evidence gaps in the understanding of cardiac issues in Duchenne.

For the last several years, PPMD has been dedicated to cardiac issues in Duchenne, launching a Cardiac Initiative in 2011. 

Heart issues don't just affect some people with Duchenne; they affect all people with Duchenne. While advances in respiratory care have improved respiratory outcomes, dilated cardiomyopathy and heart failure remain the leading cause of death in Duchenne. The goal of this first Cardiac Working Group is to identify knowledge gaps in our understanding of the natural history, diagnosis, and treatment of Duchenne-associated cardiac disease and develop a research agenda to address these gaps. We are thrilled to be collaborating with NHLBI and look forward to reporting back to the community after this week’s meeting.

-- PPMD’s Vice President of Clinical Care, Kathi Kinnett, MSN, CNP

The first meeting of the Cardiac Working Group will be structured around four topics and key questions within each topic:

1) Natural History

  • Is there an association between cardiac fibrosis and cardiac dysfunction?
  • What is the importance of genetic variants in cardiac dysfunction?
  • Is there an association between cardiac fibrosis and cardiac arrhythmias?
  • What is the true incidence of sudden cardiac death?

2) Diagnosis and Monitoring

  • What monitoring/imaging should be standard?
  • When should monitoring start?
  • What efforts are underway to advance knowledge and use of current, biomarkers and identify new cardiac biomarkers?

3) Medical Therapy

  • What is standard cardiac therapy?
  • When should it be started?
  • Are there lessons to be learned from adult heart failure?
  • How will new skeletal muscle therapies affect the heart?

4) Non-Medical Therapies

  • Are heart transplants and left ventricular assist devices viable options in Duchenne?
  • When is ICD placement appropriate?
  • Can the use of devices change the natural history of Duchenne?

The meeting will conclude with a detailed outline of next steps, recommendations, and a summary of the meeting to be published.

Funding for this meeting has been provided, in part, by John Owens Adventure, Inc., a nonprofit that funds awareness for chronic childhood illnesses, including Duchenne.

To learn more about PPMD’s Cardiac Initiative and other cardiac care issues in Duchenne, visit PPMD’s website.

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Comment by Kathi Kinnett on July 14, 2014 at 1:48pm

Frederick - 

thank you for your comment.  Through a public/pprivate partnership, PPMD was pleased to sponsor this meeting, which was organized by NHLBI.  THis was a very small, confined meeting organized to identify gaps in Duchenne cardiac care and research.  The chairs (Elizabeth McNally and Larry Markham) led 2 amazing days of discussion.  An executive summary of the meeting is forthcoming from NHLBI. The plan moving forward is the formation of 3 working groups (clinical care, human research and animal research).  Each group will summarize and update what is known in their area, and make suggestions for future research. When the chairs of the working groups are identified, you are welcome to contact the chair(s) and let them know that you are interested in participating.  We welcome your input.

Comment by Frederick Sachs on July 13, 2014 at 2:19pm

For some reason we were not invited to present anything at the  “Contemporary Cardiac Issues in Duchenne Muscular Dystrophy” meeting. We have published on both topics for years and worked with cardologists in the US and Europe on the arrhythmia issues as well as dystrophy. This is a unique peptide drug,  the only drug of its class, is non allergenic, chemically and enzymatically stable, non metabolizable, and proven to be antiarrhythmic in animals and non toxic to human heart, has  a long half life, inhibits stretch induced Ca uptake in dystrophic muscle, and is chemically synthesized at relatively low cost under GMP conditions.

It would seem that availability of a single drug that could treat MD and its associated arrhythmias could have been of interest at the meeting.

Comment by Frederick Sachs on July 13, 2014 at 2:08pm

The parents of boys with Duchenne may not be aware that we have an orphan drug for Dystrophy that is also a potent antiarrhythmic and appears totally non toxic to rabbits, mice, rats, ferrets and and humans. 

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