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A revolutionary new drug could eliminate a major genetic risk for heart disease with just one injection.
Meet lepodisiran — an experimental therapy targeting lipoprotein(a), or Lp(a), a dangerous cholesterol-like particle that's almost entirely controlled by your genes.
Unlike regular LDL ("bad") cholesterol, Lp(a) levels barely budge with diet, exercise, or standard statins. High Lp(a) is a hidden driver of heart attacks, strokes, and aortic valve disease in millions of people worldwide.
In groundbreaking early-phase clinical trials (led by teams at Stanford University and UC San Diego), a single dose of lepodisiran slashed Lp(a) levels by an astonishing 94% on average.
Even more remarkable: the reduction lasted over a year — and in many patients, levels remained dramatically suppressed even longer — after just one or two doses.
This "one-and-done" approach could completely transform heart disease prevention. Instead of lifelong daily pills, high-risk individuals might need only occasional treatments.
Larger international trials are now underway to confirm whether lepodisiran can actually prevent heart attacks and strokes in people with dangerously elevated Lp(a). If successful, it would mark one of the most significant advances in cardiovascular medicine in decades.
[Nissen, S. E., et al. Lepodisiran, an Extended-Duration siRNA Targeting Lipoprotein(a): A Phase 1 Randomized Clinical Trial. JAMA]

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