Pain in RASopathies: new investigative techniques and treatments

Awardee: Giuseppe Zampino

Institution: Università Cattolica Sacro Cuore

Award Amount: $53,000

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

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Systematic Evaluation of a Cluster of Nonsense Mutations in Exon 22 of CFTR to Inform Treatment

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