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

Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.

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. 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

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). As the first and only small interfering RNA to provide effective and sustained LDL-C reduction, Leqvio helps manage a critical cardiovascular risk factor for ASCVD. It is currently under review by the U.S. a statin or ezetimibe).

RNA 52

RNA Trials Production Containment 52

Scienmag

JULY 19, 2021

May also explain liver fat differences among men & women Credit: Sallam Lab/UCLA FINDINGS A chemical modification that occurs in some RNA molecules as they carry genetic instructions from DNA to cells’ protein-making machinery may offer protection against non-alcoholic fatty liver, a condition that results from a build-up of fat in the liver (..)

RNA 95

RNA DNA Genetics Protein 95