News
Researchers from Intellia Therapeutics, Regeneron Pharmaceuticals, and clinical partners have published the first-ever clinical data that support the safety and…
People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually…
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…
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…
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical…
Join moderators Professor Per Westermark and Professor Julian Gillmore for a three-hour seminar that will provide an overview of current methods used to diagnose…
This symposium is organized as a hybrid meeting (with the focus on in-person) held from Sunday, May 26 through Thursday, May 30, 2024.
Together with the ISA Board…
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, today announced that the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi…
Outside of specialized centers, access to the appropriate typing and studies for risk stratification as well as novel therapeutic options is still suboptimal.…
AL Amyloidosis and Agent Orange
Veterans who develop AL amyloidosis and were exposed to Agent Orange or other herbicides during military service do not have to…