amyloidosis with polyneuropathy. Like many rare diseases, hereditary ATTR is monogenic. A mutation in the TTR gene results in a misfolded protein that overwhelms the body’s capacity to clear it.

The trial has completed dosing in eight participants, including six patients with ATTR amyloidosis with polyneuropathy (ATTR-PN) and two patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM), ...

vutrisiran will become the first therapeutic approved in the United States to treat both the polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis.

The phase III MAGNITUDE 2 study is actively screening patients with hereditary ATTR amyloidosis with polyneuropathy, with the first patient expected to be dosed later in the first quarter of 2025.

ATTR is characterised by the formation of amyloid fibril deposits in tissues ... is being developed for both ATTR cardiomyopathy and polyneuropathy. The polyneuropathy indication making it a ...

Alnylam Pharmaceuticals ALNY announced preliminary net product revenues for the fourth quarter and full-year 2024 for its ...

AstraZeneca and Ionis' Wainzua has been recommended for NHS use in the treatment of patients with polyneuropathy associated with transthyretin amyloidosis (ATTR-PN). New guidance from health ...

“Genetic testing is the only way to confirm whether patients who are suspected to have hereditary ATTR amyloidosis with polyneuropathy or have a family history of the disease carry a genetic ...