Awarded Grants

The ODC offers over 50 grant opportunities in 30+ disease areas annually to researchers across the globe, as well as within the Penn and CHOP communities. Since 2011, our grant programs have funded $17.2 million in rare disease research.

See below for a complete list of ODC-awarded grants since 2011. 

Full list of 2018 MDBR Awardees

Awarded Grants

Displaying Results 1 - 10 of 168

Awardee Project Title Institution Grant Amount Funding Year
Robert Waymouth, PhD A New Concept for Gene Delivery and Therapy in Cystic Fibrosis Stanford University MDBR - Nonsense Mutations in Cystic Fibrosis $52,985 2018
Emyr Lloyd-Evans, D.Phil A humanised Drosophila melanogaster model of MPSIIIb to rapidly study central nervous system efficacy of combined chaperone and substrate reduction therapy Cardiff University MDBR - Mucopolysaccharidoses $59,449 2018
Robert Ohgami, MD A longitudinal single cell genomic analysis of HHV8-negative multicentric Castleman Disease Stanford University MDBR - Castleman Disease $42,508 2018
Suneet Agarwal, MD, PhD A multi-center, prospective, minimal intensity BMT trial for dyskeratosis congenita Boston Children's Hospital MDBR - DC $51,000 2016
Lisa Maves, PhD A zebrafish platform for DMD drug-combination testing Seattle Children's Research Institute MDBR - DMD $49,000 2015
Dolan Sondhi, PhD AAVrh.10-mediated Npc2 Gene transfer for the treatment of Npc2 Deficient Niemann-Pick disease Weill Cornell Medicine MDBR - Niemann Pick Type C $49,645 2018
Ronald Crystal, MD Adeno-associated Virus-mediated Gene Transfer for the Treatment the CNS Manifestations of MPS I Weill Medical College of Cornell University MPS I $330,000 2012
Florian Eichler, MD Adeno-associated virus serotype 9-mediated gene therapy for Adrenomyeloneuropathy Massachusetts General Hospital MDBR - ALD $50,000 2014
Joseph Anderson, PhD Alpha-L-iduronidase Delivery via Human CD34+ Hematopoietic Stem Cells to Treat Mucopolysaccharidosis Type I University of California Davis MPS I $330,000 2017
Saar Gill, MD, OhD and Brendan Weiss, MD Amyloidosis Program of Excellence Feasibility Pilot Grant University of Pennsylvania POE - Amyloidosis $60,000 2017
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