News

BREAKING NEWS: The U.S. Food and Drug Administration today approved Alnylam Pharmaceuticals AMVUTTRATM, a novel therapy for the treatment of hereditary transthyretin (hATTR) amyloidosis with polyneuropathy. This approval comes after positive results from the readout of HELIOS-A Phase III study...

The Amyloidosis Forum, a public-private partnership initiated between the Amyloidosis Research Consortium and FDA, was highlighted recently during FDA Rare Disease Day Virtual Public Meeting on March 4th as an exemplary model to enhance product development using a public-private partnership...

Rare Disease Day takes place every year on Feb 28. The many events that take place all month long provide a unique opportunity to raise awareness and advocate for key policy issues that affect amyloidosis patients. As rare diseases take...

Leading cardiologist Dr. Martha Grogan was our guest speaker for Taming Wild-Type, our recent ARC Talks webinar focused on wild-type transthyretin amyloidosis (ATTRwt). In the presentation, Dr. Grogan, the Founder and Director of the Cardiac Amyloid Clinic at the Mayo...

Stephanie Ayars’ family was forever changed after her dad received the news that he had hereditary amyloidosis. In our first post of a two-part series, Stephanie shared her dad’s story from diagnosis to organ transplants, and what led her to...