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Light-chain (AL) amyloidosis is a rare systemic disorder characterized by the deposition of misfolded protein aggregates into various organs such as the heart,…

In a letter published in HemaSphere, Claudia Bellofiore, MD, of the University of Pavia in Italy, and colleagues highlighted the need to expand clinical trial…

ABSTRACTPrimary progressive aphasia (PPA) refers to a group of clinically and pathologically heterogeneous syndromes characterized by progressive and relatively…

Attruby (acoramidis) slows disease progression and significantly reduces death and cardiovascular-related hospitalizations in adults with hereditary or wild-type…

A new entrant in the race to shepherd antibodies for Alzheimer’s disease across the blood-brain barrier has emerged. Korsana Biosciences has catapulted out of…

I often consider raising funds for patients with alpha-1 antitrypsin deficiency (AATD) like me. With February being Rare Disease Month, I recently found myself…

No FDA approved drugs exist for relapsed/refractory (RR) AL Amyloidosis. CAR-T is a novel approach. We report safety and efficacy from the first 23 pts in NEXICART-…

Background:Hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) is a progressive multisystem disorder caused by pathogenic TTR variants. Aim of this…

Join NORD’s Vermont Rare Action Network (RAN) for a Rare Disease Day advocacy event in Montpelier at the Statehouse on Thursday, February 19 from 4pm to 6pm. This…

Transthyretin amyloid cardiomyopathy (ATTR-CM) is an increasingly recognized cause of heart failure that has historically been underdiagnosed due to nonspecific…