Cardiology, Genetic Disease and Protein - Clinical Research Informer

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Cardiology

Genetic Disease

Protein

BridgeBio’s Attruby (Acoramidis): A New, More Potent TTR Stabilizer for ATTR-CM

XTalks

NOVEMBER 26, 2024

This progressive disease occurs when misfolded transthyretin (TTR) proteins form amyloid deposits in the heart, leading to cardiac dysfunction. BridgeBio has been highly active in the genetic diseases space.

Genetic Disease

Genetic Disease Genetics Protein Clinical Development

Tryngolza (Olezarsen) Becomes First FDA-Approved Drug for Familial Chylomicronemia Syndrome

XTalks

JANUARY 3, 2025

Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism. People with FCS often have triglyceride levels higher than 880 mg/dL, compared to a healthy target level of below 150 mg/dL. FCS also severely impacts quality of life, causing chronic fatigue and recurrent stomach pain.

FDA Approval

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BridgeBio’s Attruby (Acoramidis): A New, More Potent TTR Stabilizer for ATTR-CM

Tryngolza (Olezarsen) Becomes First FDA-Approved Drug for Familial Chylomicronemia Syndrome

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