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Rare Disease Day(R) takes place worldwide, typically on or near the last day of February each year, to raise awareness among policymakers and the public about rare…
The National Organization for Rare Disorders (NORD(R)), in collaboration with the Rare Disease Diversity Coalition (RDDC), is pleased to announce the second webinar…
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage genome editing company focused on developing curative therapeutics leveraging CRISPR-based…
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…
Join NHLBI, The Heart Truth(R), and organizations around the country throughout February and National Wear Red Day(R) on February 3rd, 2023 to bring greater…
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…
Investigators have found that intravenous infusion of a CRISPR-Cas9 based gene therapy decreases serum transthyretin (TTR) protein in patients with hereditary TTR…
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…