News

A single dose of Intellia Therapeutics’ investigational CRISPR/Cas9-based gene-editing therapy NTLA-2001 safely results in a rapid and strong reduction in the levels of…

Alnylam Pharmaceuticals’ application for the approval of vutrisiran, its investigational therapy for familial amyloid polyneuropathy (FAP), is being reviewed by the U.S.

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient…

Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that…

Enrollment is now completed in the Phase 3 APOLLO-B trial, which is assessing the safety and efficacy of Onpattro (patisiran) to treat heart disease…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates…

Attralus’ imaging agent AT-01 was able to detect amyloid deposits in the heart, even in patients without heart symptoms and with normal levels…

Alnylam Pharmaceuticals has initiated testing of a twice a year dosing regimen for vutrisiran, its investigational RNA-targeted therapy for familial amyloid polyneuropathy (FAP) and other forms of ATTR amyloidosis.