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Amyloidosis is a rare, chronic condition in which abnormal proteins bunch together to form amyloid deposits. These deposits can build in certain body organs such as…

Carlisle, PA…  Now in its third decade of doing good for those impacted by Amyloidosis, the Chip Miller Amyloidosis Foundation (CMAF) works year-round to raise…

NORD and Yale Genetics are hosting a Rare Disease Day celebration at the Yale Peabody Museum! Join us for a night of community building & connection. Attendees…

Join NORD’s Pennsylvania Rare Action Network for a virtual Rare Disease Day celebration on Friday, February 20 from 12pm to 1:10pm ET

Net cash used in operating and investing activities was $23.3 million and $163.7 million for the fourth quarter and full year of 2025, respectively; quarter-end…

Isatuximab monotherapy resulted in promising hematologic response rates with a good safety profile among patients with relapsed/refractory (R/R) light chain (AL)…

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…