HELIOS-B is a global, randomized, double-blind, placebo-controlled clinical trial that evaluated the efficacy and safety of AMVUTTRA (R) in adult patients with wtATTR or hATTR amyloidosis with ...

− First and Only RNAi Therapeutic Approved by the European Commission for the Treatment of Wild-Type or Hereditary ATTR Amyloidosis with Cardiomyopathy – − Novel RNAi Mechanism Delivers Rapid ...

− Vutrisiran Treatment Led to Maintenance or Improvement in Functional Capacity, Health Status, and Quality of Life, Compared to Placebo – “The HELIOS-B study continues to deliver a robust data ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference (RNAi) therapeutics company, today announced that the Committee for Medicinal Products for ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. The greatest areas of unmet needs. I think ...

It elucidates the series of events involving the dissociation, misfolding and aggregation of TTR proteins, resulting in the formation of amyloid fibrils. It also higlights various therapeutic ...

Systemic light chain (AL) amyloidosis is a rare and life-threatening disorder characterized by the deposition of misfolded immunoglobulin light chains as insoluble amyloid fibrils in various tissues ...

Impact of Azacitidine Before Allogeneic Stem-Cell Transplantation for Myelodysplastic Syndromes: A Study by the Société Française de Greffe de Moelle et de Thérapie-Cellulaire and the ...

− Phase 3b Open-Label Study Showed Treatment with Patisiran Achieved Rapid and Sustained Reduction in Serum TTR Levels in hATTR Amyloidosis Patients with Polyneuropathy Progression Following ...

Alnylam Canada ULC is pleased to announce that Health Canada has issued a Notice of Compliance (NOC) authorizing AMVUTTRA® (vutrisiran) for the treatment of cardiomyopathy in adult patients with ...

ATTR-CM is a debilitating, rapidly progressive and potentially fatal disease for which there are limited treatment options. Vutrisiran is currently approved in the European Union (EU) under the brand ...