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 105

Genetic Disease Genetics Protein Clinical Development 105

Scienmag

NOVEMBER 18, 2020

— Research on a genetic heart disease has uncovered a new and unexpected mechanism for heart failure. This landmark discovery found a correlation between the clumping of RNA-binding proteins long linked to neurodegenerative disease ? ROCHESTER, Minn.

Genetics 40

Genetics RNA Protein Research 40

XTalks

SEPTEMBER 4, 2024

The data, presented at the European Society of Cardiology Congress 2024, suggest that vutrisiran could significantly reduce the risk of death and heart-related issues, marking a potential treatment breakthrough. Vutrisiran works by silencing the gene responsible for producing TTR, reducing both mutant and wild-type TTR proteins.

Trials 52

Trials RNA Protein Gene Editing 52

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. Tryngolza works by targeting a protein in the liver, apoC-III, which regulates triglyceride metabolism.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

Scienmag

OCTOBER 7, 2020

Collaborative projects funded by the awards will address important questions about neurodegenerative disease, nucleus biology, and protein turnover Credit: Dollie LaJoie, Huntsman Cancer Institute at the University of Utah Could scientists detect neurodegenerative diseases like Alzheimer’s and ALS years or decades before these disorders manifest (..)

Protein 52

Protein Scientist Research Cardiology 52

The Pharma Data

DECEMBER 8, 2020

The company’s commercial priorities are three-fold: (1) Initially focusing its commercial efforts on rare diseases within its prolific neurology and cardiology franchises (2) pioneer new markets where there are no available treatments (3) create new standards of care where there has been a lack of innovation to optimize patient care. .

Medicine 52

Medicine RNA Production Marketing 52

The Pharma Data

AUGUST 30, 2021

Results were presented at the ESC Congress 2021, organized by the European Society of Cardiology (ESC). Leqvio works differently from other therapies by preventing the production of the target protein in the liver, increasing hepatic uptake of LDL-C and clearing it from the bloodstream 17. a statin or ezetimibe).

RNA 52

RNA Trials Production Containment 52