A pill helps teens with a thickened heart, easing a blockage that once needed surgery
In teens with a thickened heart muscle, a daily pill called mavacamten sharply reduced a blockage in blood flow leaving the heart. After 28 weeks, the blockage pressure dropped by about 48 units in the pill group versus almost nothing on a dummy pill.
Why it matters
Hypertrophic cardiomyopathy is when the heart muscle grows too thick, which can block blood from leaving the heart and cause fainting or breathlessness. Until now, young patients often faced surgery because no approved medicine existed for them. This trial suggests a pill could offer a gentler option. It was small, so larger and longer studies are still needed before doctors know the full picture.
Who's behind it: Joseph W. Rossano and colleagues, The SCOUT-HCM trial, an international team led from children's heart centers. Funded by Bristol Myers Squibb, which makes the drug being tested.
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