News
The Rare Disease Day Symposium at the Quinnipiac Frank H. Netter MD School of Medicine is unique to the state of Connecticut in addition to a global event that…
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…
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…