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Express the first approved therapy!

 Last Update: 2022-09-07
 Source: Internet
 Author: User

▎Edited by WuXi AppTec Content Team

Today, the U.

People with ASMD lack the enzyme that breaks down sphingomyelin, which results in the accumulation of this complex lipid molecule in the liver, spleen, lungs, and brain, causing abdominal enlargement and causing pain, vomiting, difficulty eating, and ease Symptoms such as falls

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References:

[1] FDA Approves First Treatment for Acid Sphingomyelinase Deficiency, a Rare Genetic Disease.

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Express the first approved therapy!

▎Edited by WuXi AppTec Content Team

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Express U.S. FDA approves the first thymic tissue regeneration therapy