Amyloidosis, like Alzheimer’s, results in a build up of misfolded proteins in the body. However, amyloidosis affects the rest of the body, varying by patient, while Alzheimer’s is found to only deposit the proteins in the brain.
Intellia Pharmaceuticals presented at the JPM Conference in San Francisco last month, focusing on some incredibly exciting drugs. Below are the highlights of the presentation. Nex-z, formerly known as NTLA-2001, may provide a single dose cure to what has historically been a terminal rare disease, Transthyretin amyloidosis (ATTR).
Until 2019 there was no treatment for cardiac amyloidosis. Then Vyndamax was introduced and has stabilized the disease for many. In November 2024, Attruby was approved as a superior stabilizer.
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