Pilot Study of the Efficacy and Safety of Sirolimus in the Treatment of Congenital Hyperinsulinism
Awardee: Diva De Leon
Institution: Children's Hospital of Philadelphia
Award Amount: $60,000
Funding Period: January 1, 2015 - December 31, 2015
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Adeno-associated virus serotype 9-mediated gene therapy for Adrenomyeloneuropathy
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