News

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

Amyl Therapeutics has raised €18.3 million — about $22.2 million — to develop its ClariTY technology platform to advance therapeutic candidates for familial amyloid polyneuropathy (FAP) and other forms of amyloidosis. “The ClariTY platform offers the unique opportunity to generate therapeutic candidates able to target all amyloid fibril…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

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 in people with transthyretin-mediated (ATTR) amyloidosis, a group of conditions that includes familial amyloid polyneuropathy (FAP). The study (NCT03997383) has enrolled more than…

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 of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Attralus’ imaging agent AT-01 was able to detect amyloid deposits in the heart, even in patients without heart symptoms and with normal levels of a heart damage biomarker, according to data from a Phase 1/2 clinical trial. The imaging agent was also able to distinguish between patients with…

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. The new dosing regime is being added to the open-label extension phase of the ongoing Phase 3 HELIOS-A trial (NCT03759379) that is now underway…

Living with hereditary transthyretin amyloidosis (hATTR), a group of rare disorders that includes familial amyloid polyneuropathy (FAP), affects the quality of life for both patients and their families, a large questionnaire study in Italy found. Among the more common and practical consequences for…

Editor’s note: The FAP News Today team provided in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference. There are roughly 2,700 people in the U.S. with hereditary transthyretin (ATTRv) amyloidosis, a group of rare disorders that also…