A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with ...

Based on Nobel Prize-winning CRISPR/Cas9 gene editing technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate ...

The hereditary transthyretin amyloidosis market is experiencing significant growth driven by increasing disease awareness, ...

With targeted treatment using both medical therapy for transthyretin-associated cardiac amyloidosis (ATTR-CA) and valve replacement for aortic stenosis (AS), patients with the dual pathology can fare ...

AL (immunoglobulin light chain) amyloidosis is a rare disease that often results in progressive organ dysfunction, organ failure and eventual death. Clonal plasma cells in the bone marrow secrete free ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. The greatest areas of unmet needs. I think ...

ALSO TESTED POSITIVE. A LOCAL EFFORT TO FIND AND TREAT A HIDDEN HEART DISEASE. SOME EXPERTS REFER TO IT AS STIFF HEART SYNDROME, BUT FEW KNOW AS MUCH ABOUT THE SYMPTOMS. AS DOCTORS AT BRIGHAM AND ...

Owing to the low incidence of AL amyloidosis, large studies are scarce and hardly any randomized, controlled studies comparing different therapeutic regimens have been conducted. Comparison of studies ...

The market size for hereditary transthyretin amyloidosis in the leading markets is expected to grow significantly by 2034. The United States accounted for the highest hereditary transthyretin ...

CAMBRIDGE, Mass., Sept. 22, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based ...