The Amyloidosis Forum: a public private partnership to advance drug development in AL amyloidosis.

ABSTRACT:

Background

Immunoglobulin light chain (AL) amyloidosis is a rare, multi-systemic disorder characterized by two disease processes: an underlying plasma cell dyscrasia that provides the source of pathologic light chains, and the resulting organ dysfunction caused by deposition of amyloid light chain fibrils. There are no FDA approved treatments for AL amyloidosis; regimens developed for multiple myeloma are used off-label to treat the plasma cell disorder and no therapies are directed at organ deposition. Thus, an unmet medical need persists despite advances in disease management. A public-private partnership was recently formed between the Amyloidosis Research Consortium (ARC) and the US Food and Drug Administration (FDA) to bridge scientific gaps in drug development for the treatme

SUBMITTER: Inaugural Amyloidosis Forum Panelists 

PROVIDER: S-EPMC7523334 | biostudies-literature | 2020 Sep

REPOSITORIES: biostudies-literature