RARE publishes GENGLYCOS 96-week GSDIa trial in Journal of Inherited Metabolic Disease
Ultragenyx (RARE) announced publication of a 96-week randomized, placebo-controlled trial of GENGLYCOS (DTX401) AAV gene therapy in GSDIa with crossover treatment.
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08:07:39 PM UTC
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At Week 96, participants across treatment and crossover groups experienced mean reduction in daily cornstarch intake of 61% while maintaining glycemic control, with most participants achieving reduction of at least 50%Complete elimination of nighttime cornstarch dosing observed in 33% of DTX401 tre…
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