Familial Amyloidosis: “So Rare You’ll Never See Anyone With It”

Familial Amyloidosis: "So Rare You’ll Never See Anyone With It" By Kathleen Hoffman, PhD, MSPH Until recently, the only treatments for a rare disease called familial amyloidosis were symptom management or a liver transplant. Not only is the disease inherited, genetic, and rare, its incidence varies widely by region: The NIH says it occurs in about 1 in 583 people in some parts of Portugal, compared to about 1 in 100,000 people in the US. Inspire members say that the path to diagnosis and treatment is long and convoluted: I was diagnosed with familial TTR Amyloidosis about [...]