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 ...

TTR protein is produced primarily in the liver and is normally a carrier of vitamin A. Mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as 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 ...

About ATTR Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid ...

− 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 ...

Alnylam Pharmaceuticals Inc. ALNY said Wednesday its stock will be halted on Nasdaq through the day as a regulatory advisory committee will meet to review its new drug application for a treatment for ...

"Our data support the use of sNfL in screening TTRv carriers and in monitoring disease progression and effect of treatment in ATTRv amyloidosis patients…Larger studies are warranted to confirm and ...

Near-complete in vitro TTR stabilization was observed as early as day 28 and through completion of a 30-month study of patients with ATTR-CM. The Food and Drug Administration (FDA) has approved ...

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-For each 5-mg/dL increase in serum TTR level within 28 days of starting treatment, the relative risk reduction of mortality was up to 31.6% through Month 30, confirming the hypothesis that ever ...

CAMBRIDGE, Mass., April 03, 2025 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, ...

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 ...