Same applies to cancer which has been successful. How is transthyretin different ?
March 4
Is there work on fixing or rebuilding the transthyretin protein for a cure?
Currently, available treatments focus on managing amyloidosis rather than curing it by fixing the protein itself. The main approaches work in different ways:
Stabilizers like tafamidis and acoramidis keep the TTR protein in its correct shape to Show Full Answer
Is there work on fixing or rebuilding the transthyretin protein for a cure?
Currently, available treatments focus on managing amyloidosis rather than curing it by fixing the protein itself. The main approaches work in different ways:
Stabilizers like tafamidis and acoramidis keep the TTR protein in its correct shape to prevent clumping, but they don't repair or rebuild the protein.
Gene-silencing therapies like patisiran, vutrisiran, and eplontersen reduce how much abnormal TTR protein your liver makes in the first place. These use RNA interference technology to "silence" the faulty gene.
While researchers are exploring combination therapies that use both stabilizers and gene-silencing drugs together, the current treatments require lifelong use. The protein is constantly being made by your body, so stopping treatment would allow abnormal protein to build up again.
The focus right now is on early diagnosis and starting treatment before significant organ damage occurs, which leads to better outcomes. New biomarkers and improved testing are helping doctors track treatment effectiveness more closely.
March 4
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