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$RARE Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia https://globenewswire.com/news-release/2019/01/04/1680634/0/en/Ultragenyx-Announces-Positive-Topline-Results-from-First

08:34 EST 4 Jan 2019 | Odi Bruckman

Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia https://globenewswire.com/news-release/2019/01/04/1680634/0/en/Ultragenyx-Announces-Positive-Topline-Results-from-First-Cohort-of-Phase-1-2-Clinical-Study-of-DTX401-Gene-Therapy-in-Glycogen-Storage-Disease-Type-Ia.html …

Original Article: $RARE Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia https://globenewswire.com/news-release/2019/01/04/1680634/0/en/Ultragenyx-Announces-Positive-Topline-Results-from-First

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Gene Therapy
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...