Natural history study and biomarker development in the large animal model of mucolipidosis II

Awardee: Charles Vite

Institution: University of Pennsylvania

Award Amount: $50,500

Funding Period: January 1, 2016 - December 31, 2016

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Pain in RASopathies: new investigative techniques and treatments

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Targeted AAV gene therapy for vertical supranuclear gaze palsy and dysphagia