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Cellular pathway of genetic heart disease similar to neurodegenerative disease

Scienmag

— 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.

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Vutrisiran Reduces Heart Risks for ATTR-CM Patients in Alnylam’s HELIOS-B Trial

XTalks

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.

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Ionis highlights achievements, commercial strategy and technology advancements at Investor Day

The Pharma Data

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.

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Novartis Leqvio®* (inclisiran) analyses show effective and sustained LDL-C reduction.

The Pharma Data

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).

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RNA modification may protect against liver disease

Scienmag

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 (..)

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