Alnylam announced the FDA approval of its supplemental new drug application for vutrisiran, an RNA interfering small molecule ...

Amyloidosis is the basis of several serious diseases, such as Alzheimer's disease and Parkinson's disease. This process involves the formation of amyloid fibrils, crystal-like collections of ...

Amyloidosis is the basis of several serious diseases, such as Alzheimer disease and Parkinson disease. This process involves the formation of amyloid fibrils, crystal-like collections of misfolded ...

Impact of Baseline Heart Failure Severity on Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy in the HELIOS-B Trial: A Subgroup Analysis An exploratory ...

Protein folding is the process by which proteins achieve their ... The pathophysiology of Amyloid light-chain (AL) amyloidosis remains poorly understood due to the lack of reliable in vivo models.

AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR) a key component of its rare disease pipeline, licensing a second drug candidate in the space of a couple of months.

Washington University School of Medicine led a study evaluating long-term gantenerumab treatment in individuals with dominantly inherited Alzheimer's disease (DIAD). Gantenerumab was associated with ...

The RMAT designation could expedite the development and approval process, offering Immix Biopharma ... the safety and efficacy of NXC-201 in AL amyloidosis patients. NXC-201 is the only CAR ...

in which amyloid β-protein oligomers adversely affect synaptic structure and plasticity. Findings in other neurodegenerative diseases indicate that a broadly similar process of neuronal ...