Once-daily Pill Could Help Teens with Common Genetic Heart Disease

A Philadelphia researcher finds a drug already approved for adults may also significantly reduce blockages in youth with hypertrophic cardiomyopathy.

Apr. 9, 2026 at 9:41pm

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A translucent X-ray image of an enlarged human heart, with the internal structures visible as glowing lines and shapes, representing the effects of hypertrophic cardiomyopathy on the organ.A new medication could help teens with a common genetic heart condition avoid invasive surgery.Philadelphia Today

A once-daily pill could change how doctors treat teens with hypertrophic cardiomyopathy, a genetic heart condition that can block blood flow, a Philadelphia researcher has found. Children's Hospital of Philadelphia cardiologist Joseph Rossano led an international trial involving 44 patients ages 12 to 17 that studied a drug already approved for adults with the condition. His work showed the drug, mavacamten, could also significantly reduce blockages in youth and possibly delay the need for surgery.

Why it matters

Hypertrophic cardiomyopathy is a common inherited heart disease, estimated to affect one in 500 Americans. The condition causes the heart muscle to thicken, which can obstruct blood flow and lead to heart failure, abnormal heart rhythms, and in rare cases, sudden cardiac death. Finding a medication that can effectively treat this condition in teens could help avoid the need for invasive surgery.

The details

In the trial, teens were randomly split into two groups - one receiving mavacamten and the other a placebo. After 28 weeks, the teens receiving the drug saw their obstruction levels cut substantially, by an average of 48 mmHg, compared to just 0.5 mmHg in the placebo group. The results suggest the drug could delay or potentially avoid the need for surgery in many young patients.

  • The trial's findings were presented at the American College of Cardiology's annual scientific session in New Orleans and simultaneously published in The New England Journal of Medicine in April 2026.

The players

Joseph Rossano

A cardiologist at Children's Hospital of Philadelphia who led the international trial studying mavacamten in teens with hypertrophic cardiomyopathy.

Elizabeth Blume

A pediatric cardiologist and professor of pediatrics at Harvard Medical School who was not involved in the study but provided independent analysis.

Bristol Myers Squibb

The pharmaceutical company that manufactures mavacamten and financially supported the study.

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What they’re saying

“Imagine being the parent of a 12-year-old who can now take a medicine by mouth rather than have open heart surgery.”

— Elizabeth Blume, Pediatric Cardiologist and Professor of Pediatrics at Harvard Medical School

“If we identify patients young with this and get started on medicines early, their hearts could be much better, 10, 20, 30 years later than if they weren't started on the medicine.”

— Joseph Rossano, Cardiologist, Children's Hospital of Philadelphia

What’s next

Longer-term studies are being planned to further evaluate the effects of mavacamten in teens with hypertrophic cardiomyopathy and determine if the drug can avoid the need for surgery altogether.

The takeaway

This study represents a significant milestone in the treatment of hypertrophic cardiomyopathy in young patients, potentially offering a non-surgical option that could preserve heart function and improve long-term outcomes.