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Intellia and Ultragenyx’s Clinical Updates, Novo Holdings’ Catalent Acquisition, and NanoString Bankruptcy; Simon Barnett

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Gene Therapy for Pediatric Neurodegenerative Disorders

This chapter explores the clinical trials conducted by UltraGenyx on gene therapy for pediatric neurodegenerative disorders, specifically mucopolysaccharidoses. The trial shows promising results with significant improvements in cognitive function and a decrease in toxic substances related to the disease.

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