Cardiomyopathy | Research and Clinical trials

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Contact the Cardiomyopathy Program

At Boston Children's Hospital, our care is informed by our research, and our discoveries in the laboratory strengthen the care we provide at each child's bedside.

Boston Children’s scientific research program is one of the largest and most active of any pediatric hospital in the world. In particular, our cardiovascular and cardiac surgery researchers are yielding crucial insights into the causes and development of heart disease and disorders; paving ground for the most promising new treatments and interventions; and offering ideas and discoveries that could one day lead to cures.

Among our current research projects with promise for treating cardiomyopathy are: 

Understanding family ties to hypertrophic cardiomyopathy

Working with colleagues at Brigham and Women’s Hospital, Children’s researchers are examining common characteristics among families with a genetic predisposition toward hypertrophic cardiomyopathy. The team is leading a multi-center registry of children and adults who carry the gene for this type of cardiomyopathy, in hopes that studying their genetic makeup will help them identify the exact mutations influencing the onset of the disease.

Reversing heart damage with a simple injection

A team of Boston Children’s researchers—led by Bernhard Kuhn, MD, of the Department of Cardiology—have demonstrated that a growth factor called neuregulin1 (NRG1) can encourage the growth of muscle and the restoration of function after damage to the heart. Mice who received a simple injection of NRG1 have experienced considerable reversal of pre-existing damage caused by heart attacks.

Evaluating the effectiveness of medication in delaying hypertrophic cardiomyopathy

Researchers in Boston Children’s Department of Cardiology and at Brigham and Women’s Hospital are conducting medication trials for patients who carry the gene for hypertrophic cardiomyopathy, but whose echocardiograms are normal. The aim of the project is to develop new drug therapies capable of delaying the onset of symptoms.

Clinical trials

Boston Children’s is known for pioneering some of the most effective diagnostic tools, therapies and preventive approaches in pediatric cardiology. A significant part of our success comes from our commitment to research—and to advancing the frontiers of mental health care by conducting clinical trials.

Boston Children’s coordinates hundreds of clinical trials at any given time. Clinical trials are studies that may involve:

  • evaluating the effectiveness of a new drug therapy
  • testing a new diagnostic procedure or device
  • examining a new treatment method for a particular condition
  • taking a closer look at the causes and progression of specific diseases

Boston Children’s is involved in several multi-site clinical trials and studies focusing on pediatric cardiology and cardiac surgery, in particular. While children must meet strict criteria in order to be eligible for a clinical trial, your child may be eligible to take part in a study. Before considering this option, you should be sure to:

  • consult with your child’s treating physician and treatment team
  • gather as much information as possible about the specific course of action outlined in the trial
  • do your own research about the latest breakthroughs relating to your child’s condition

If your physician recommends that your child participate in a clinical trial, you can feel confident that the plan detailed for that study represents the best and most innovative care available. Taking part in a clinical trial at Boston Children’s is entirely voluntary. Our team will be sure to fully address any questions you may have, and you may remove your child from the medical study at any time.

The role of stem cells in the fight against heart disease. Learn more about our research in this vital area.

Follow pediatric care's long history in innovation

In 1938, Boston Children’s cardiac surgeon Robert Gross, MD, performed the world’s first successful surgery to correct a child’s heart defect. Since that time, Boston Children’s has gained recognition around the globe for our leadership in pediatric cardiology, and we continue to make critical advances in the field. Follow our timeline of Boston Children’s innovations in heart care.

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

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