“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 ...

Long-term treatment with patisiran (Onpattro) led to modest changes for people with hereditary transthyretin amyloidosis ...

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.

AstraZeneca and Ionis are set fair for EU approval of Wainzua, their treatment for polyneuropathy associated with transthyretin-mediated amyloidosis (ATTR), after the EMA's human medicines ...

Compared with AS alone, coexisting ATTR-CA was associated with an increased risk of mortality (HR 1.3, 95% CI 1.1-1.4) in a ...

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 ...

Intellia doses the first patient in the phase III HAELO study on NTLA-2002 for treating hereditary angioedema. Stock rises.

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), ...

Its marketed products include ONPATTRO (patisiran) for the treatment of the polyneuropathy of hereditary ... the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy ...

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.