Systemic amyloidoses are potentially lethal ... amyloidosis and intensive chemotherapy in immunoglobulin light chain (AL) amyloidosis. These therapies often elicit substantial morbidity and ...

According to Alnylam’s Tim Mooney, mivelsiran’s endurance is due to stabilizing tweaks the scientists made to the siRNA ...

Preclinical studies have shown AT-02 is able to bind to multiple amyloid types and can induce macrophage mediated amyloid phagocytosis and removal. The Food and Drug Administration has granted Orphan ...

BridgeBio Pharma (BBIO) wins FDA nod Attruby for rare heart disorder, ATTR-CM challenging Pfizer's (PFE) Vyndaqel. Read more ...

This important study identifies the "H-state" as a potential conformational marker distinguishing amyloidogenic from non-amyloidogenic light chains, addressing a critical problem in protein misfolding ...

Amyloidosis is a rare disease characterized by extracellular protein deposits in various tissues and vital organs of the body, which often leads to severe and debilitating chronic health problems ...

Results for the Phase I clinical trial, published in the New England Journal of Medicine, bring hope for people with transthyretin amyloidosis (ATTR), who have a build-up of misfolded transthyretin ...

(RTTNews) - Alnylam Pharmaceuticals Inc. (ALNY) has shared new findings from its Phase 1 study of nucresiran (previously known as ALN-TTRsc04), a next-generation RNAi therapy being developed for the ...

Alnylam Pharmaceuticals ALNY presented new data from its early-stage study of nucresiran (formerly ALN-TTRsc04) for treating ...

The study showed significant reductions in serum TTR levels in subjects after a single dose, with sustained effects over time ...

Diagnosis of hereditary amyloid transthyretin (hATTR) amyloidosis with cardiomyopathy is frequently delayed, in large part because of symptom overlap with other cardiovascular diseases and limited ...