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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…

Immunoglobulin light chain (AL) amyloidosis is a rare and potentiallylife‐threatening disorder characterized by the deposition ofmisfolded, toxic immunoglobulin…

Biology of sex differencesBiol Sex Differ. 2026 Feb 17. doi: 10.1186/s13293-025-00800-w. Online ahead of print.ABSTRACTAlzheimer's disease (AD) is a progressive…