Establishing a Krabbe Disease Newborn Screening Biobank and Natural History Database Platform

Awardee: Laura Adang

Institution: Children's Hospital of Philadelphia

Grant Amount: $150,000

Funding Period: August 1, 2023 - July 31, 2024

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Development of a validated and patient-specific GNAO1 model for screening and testing of therapeutics

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Testing mechanisms and intervention strategies in GNAO1 dystonia