XTalks

NOVEMBER 26, 2024

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.

Genetic Disease 105

Genetic Disease Genetics Protein Clinical Development 105

Scienmag

FEBRUARY 17, 2021

A leading cause of SCD in young athletes is arrhythmogenic cardiomyopathy (ACM), a genetic disease in which healthy heart muscle is replaced over time […]. Credit: Mark Bauer/FSU College of Medicine Nearly a half-million people a year die from sudden cardiac death (SCD) in the U.S.

Medicine 55

Medicine Genetic Disease Development Genetics 55

XTalks

APRIL 15, 2021

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. Figure 1: The use of Mendelian randomization to validate genetic drug targets.

Development 98

Development Drugs Genomics Genome 98

XTalks

JANUARY 3, 2025

FCS is a rare genetic disorder that prevents the body from properly breaking down triglycerides (a type of fat in the blood), leading to dangerously high levels. This can result in acute pancreatitis (inflammation of the pancreas), a life-threatening condition causing severe abdominal pain and other health complications.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59