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

Alnylam Announces FDA Approval of AMVUTTRA™ (Vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration (FDA) approved AMVUTTRA(tm) (vutrisiran…

Alnylam Presents New 18-Month Results From Exploratory Cardiac Endpoints in HELIOS-A Phase 3 Study of Investigational Vutrisiran

Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced new positive results from an 18-month analysis of exploratory cardiac…

BridgeBio Pharma Presents Updated Results From Phase 2 Open-Label Extension Study of Acoramidis in Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

BridgeBio Pharma, Inc., a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced updated data from its ongoing Phase 2…

Positive New Data from Phase 1/2 Trial of AT-01 Presented at 2022 American College of Cardiology Annual Scientific Session

Attralus, Inc., a clinical stage biopharmaceutical company developing transformative medicines to improve the lives of patients with systemic amyloidosis, today…

First Gene-Editing Treatment Injected Into the Blood Reduces Toxic Protein for up to 1 Year

Intellia and Regeneron Announce Updated Phase 1 Data Demonstrating a Single Dose of NTLA-2001, an Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis, Resulted in Rapid, Deep and Sustained Reduction in Disease-Causing Protein

Intellia Therapeutics to Present at the 40th Annual J.P. Morgan Healthcare Conference on Wednesday, January 12, 2022

As Amyloidosis Field Explodes, Physicians Need to Spot and Treat

Cardiac amyloidosis is not nearly as rare as one might think, and with effective treatments finally emerging in recent years, there's a need to increase awareness…

UK Approves Phase 1 Trial of Potential Gene Editing Therapy, NTLA-2001

The treatment uses CRISPR/Cas9 editing technology, a technique derived from bacteria that allows researchers to edit the genome of a cell by specifying regions of…

New Era of Medicine: Researchers Publish First Positive Clinical Data for In Vivo Genome Editing in Humans

Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and…

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RNAi Explained

Dr. Bersell explores RNA interference (RNAi) technology and its applications in treating ATTR-CM and ATTR-PN. Learn about mechanism of action, clinical evidence, and patient outcomes.

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Expert Perspectives on Attruby®

Dr. Barry Trachtenberg and ATTR community members discuss Attruby™ (acoramidis), covering clinical data, patient perspectives, and treatment considerations.

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