Quantitative description of the clinical course of Canavan disease

Awardee: Annette Bley

Institution: University Medical Center Hamburg-Eppendorf, Dept of Pediatrics

Award Amount: $35,000

Funding Period: January 1, 2015 - December 31, 2015

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Dual targeting of defective lipid and metal pathways in Niemann-Pick type C disease: an in vitro pilot study

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Evaluation of PLK1 inhibitors in a pre-clinical LAM animal model