First Generation Pharmaceutical Treatments for Transthyretin Amyloidosis Continue to Make Progress

Perhaps a score of the countless proteins in the human body misfold in large amounts in later life. The misfolded form is insoluble, leading to solid deposits of the protein in and around cells. These problem proteins are known as amyloids, and the accumulation of amyloids is one of the root causes of aging. Amyloidosis conditions arise from the presence of amyloid and the disruptive effect it has on cellular biochemistry. The best known form of amyloid is the amyloid-β thought to cause Alzheimer’s disease, but the research community is beginning to appreciate that other forms may be just as big a problem over the course of aging. The topic for today is transthyrein amyloid, and the efforts to produce therapies to address it and its consequences.

misfoldamyloidsaccumulation of amyloidsAmyloidosisamyloid-βAlzheimer’s diseasetransthyrein amyloid

Transthyretin, or TTR, amyloid is of particular note in the cardiovascular system. Evidence suggests that transthretin amyloidosis is the full stop at the upper end of the natural human life span; the majority of the tiny fraction of people who survive everything else that aging can throw at the human form, and manage to become supercentenarians living to age 110 or later, will die because TTR amyloid clogs their cardiovascular

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