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

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 ...

Researchers developed trehalose-functionalized peptide amphiphiles (TPAs) that self-assemble into supramolecular ...

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 ...

About 150 people in Singapore have transthyretin amyloid cardiomyopathy. Read more at straitstimes.com. Read more at ...

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

Treatment modalities include liver transplantation for transthyretin-related (ATTR ... indispensable in the diagnosis and follow-up of AL amyloidosis. Manipulation of amyloid deposition and ...

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 ...

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 ...

called Amvuttra (vutrisiran), is made by Alnylam Pharmaceuticals and is used to treat transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a disease in which harmful proteins build up in the ...

today announced positive two-year follow-up data from the ongoing Phase 1 trial of investigational nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy ...