PMM2-CDG can lead to death in 20% of patients in the first 4 years of life, likely due to both neurological issues and multi-organ failure. While some individuals who survive to childhood have a good life expectancy, they also experience disability and typically need lifelong assistance.

Given the severity of symptoms across all stages of PMM2-CDG, significant unmet need exists for nearly all patients.

AT-007-1005 Clinical Study

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