Clinical Trials and Research

Change point analysis of the total PYP orders showed a significant difference in the number of PYP orders

Increasing clinicians’ suspicion of ATTR amyloidosis using a retrospective algorithm

Gene Editing Therapy Paves Way for Revolution in Treatment of Genetic Disorders

People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually…

CRISPR Injected Into the Blood Treats a Genetic Disease for First Time

The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an…

Landmark CRISPR Trial Shows Promise Against Deadly Disease

Preliminary results from a landmark clinical trial suggest that CRISPR-Cas9 gene editing can be deployed directly into the body to treat disease. The study is the…

Alnylam Reports Positive Topline 18-Month Results From HELIOS-A Phase 3 Study of Vutrisiran in Patients With hATTR Amyloidosis With Polyneuropathy

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi…

CRISPR Gene Editing Reduces Disease-Causing Protein in Hereditary Transthyretin Amyloidosis

The results of the phase 1 study open-label trial showed the therapy was safe, raising the possibility of a more effective and more appealing treatment for the…

FDA Approves Subcutaneous Daratumumab Plus VCd for Newly Diagnosed Light-Chain Amyloidosis

Alnylam Reports Positive Topline Results From HELIOS-A Phase 3 Study of Vutrisiran in Patients With hATTR Amyloidosis With Polyneuropathy

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced today that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi…

Janssen Submits Application Seeking U.S. FDA Approval of DARZALEX FASPRO™ (Daratumumab and Hyaluronidase-fihj) for the Treatment of Patients With Light Chain (AL) Amyloidosis

National Study Seeks Earlier Diagnosis of ATTR Cardiac Amyloidosis in Minorities

Researchers at Boston Medical Center (BMC) and Boston University School of Medicine (BUSM), in collaboration with Columbia University Irving Medical Center (CUIMC…

Alnylam Pharmaceuticals Announces Initiation of APOLLO-B Phase 3 Study of Patisiran for the Treatment of Transthyretin Amyloidosis with Cardiomyopathy

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company has initiated APOLLO-B, a global Phase 3…

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