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

XTalks

For the first time in the US, adults living with familial chylomicronemia syndrome (FCS) have an FDA-approved treatment option.

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

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BridgeBio has been highly active in the genetic diseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.

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HFpEF vs. HFrEF: How To Improve Heart Failure Drug Development

XTalks

Dr. Benoit Tyl, MD, FESC, medical and scientific director, cardiology at Servier, says that the disconnect between correlation and causality in cardiovascular disease trials is partially responsible for the lack of approved HFpEF therapies. However, these drugs have shown limited effectiveness in treating HFpEF.