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FDA accelerates approval of the first achondroplasia treatment for the root cause of the disease

 Last Update: 2021-12-02
 Source: Internet
 Author: User

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On November 19, 2021, BioMarin Pharmaceutical announced that the U.

Achondroplasia is mainly manifested by the slow growth of the bones at the base of the limbs, spine and skull.

Its pathogenesis is due to mutations in the gene encoding fibroblast growth factor receptor 3 (FGFR3), resulting in overactive FGFR3 protein

▲In the process of normal bone growth, chondrocytes will continue to form new bones on the growth plate

This approval is based on the positive results of a phase 3 clinical trial and its open-label extension study

"Chondroplasia is a lifelong genetic disease caused by bone structural disorders caused by impaired

Reference materials:

[1] BioMarin Receives FDA Approval for VOXZOGO™ (vosoritide) for Injection, Indicated to Increase Linear Growth in Children with Achondroplasia Aged 5 and Up with Open Growth Plates.

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