which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious ...

Axsome Therapeutics (NASDAQ: AXSM), and Summit Therapeutics (NASDAQ: SMMT). (Alnylam Pharmaceuticals): In her comments ...

Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity ...

Let’s cover some of the most serious, yet silent, symptoms of ATTR-CM you NEED to know and what you can do to protect ...

Long-term data from the 42-month open-label extension demonstrated that maintaining stable TTR levels significantly lowered ...

Intellia's CRISPR therapy nexiguran ziclumeran maintained reductions in the levels of a misfolded protein in the nerves of ...

While the results bolster efficacy, questions remain about high costs and other issues with the ATTR-CM therapies.

An early increase in serum TTR levels on Day 28 of dosing was associated with reduced all-cause mortality (ACM) in univariate analysis, an association which persisted in multivariate analysis ...

AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR ... Ionis that gave it rights to eplontersen, a follow-up to Ionis' already-marketed Tegsedi (inotersen) for ...

The FDA approved a test that can help clinicians diagnose Alzheimer's disease early on using a simple blood draw. The test ...

Intellia Therapeutics announced positive two-year follow-up data from its Phase 1 trial of nexiguran ziclumeran (nex-z) for treating hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN).