For decades, AL amyloidosis was a disease for which there were no FDA-approved therapies, treatments we did have were often borrowed from multiple myeloma, and resulted in poor outcomes for patients. It is one of the 7,000 diseases classified as a “rare disease,” and over 95% of those have no FDA-approved drug. In 2018 that all changed and it created a cascade of drug approvals, accompanied by a flourishing pipeline of diverse clinical trials. This has launched a world of accelerated research on amyloidosis, giving hope to patients. In today’s episode we’ll be talking with Dan about “hope” and how life with amyloidosis is brighter than ever before. For an overview of amyloidosis, please see episode 2 (for clinicians) or episode 3 (for patients).
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