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World Acromegaly Day: ASO drugs show efficacy

 Last Update: 2021-12-09
 Source: Internet
 Author: User

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Today ASO manufacturer Ionis announced a phase II clinical result of acromegaly of its liver-targeted growth hormone receptor (GHR) trans-nucleic acid drug Cemdelirsen (IONIS-GHR-L)

Drug source analysis

Today is World Acromegaly Day.

Cemdelirsen is a GalNac-coupled ASO, so it only has a significant effect on liver GHR

ASO is a treatment with a longer history than RNAi and mRNA, but because the delivery problem has not been solved, progress has been slow in the first two decades

The discovery of GalNac completely changed the fate of RNA drugs, but also restricted the target to the liver.

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