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 31 - 40 of 168

Awardee Project Title Institution Grant Amount Funding Year
Don Mahuran, PhD Developing Substrate Reduction Therapy for MPS I, II & III The Hospital for Sick Children MPS I $140,650 2013
Lorne Clarke, MD Developing Substrate Reduction Therapy for MPS I, II & III University of British Columbia MPS I $140,650 2014
Jan Nolta, PhD Development of TALE and CRISPR/Cas9 as a putative therapy for treatment or correction of genetically linked CDKL5 Deficiency University of California, Davis Medical Center CDKL5 $300,000 2017
Robin Kleiman, PhD / Michael Johnson, MD, PhD Development of a High-content drug repurposing screen using CDKL5 patient derived iPSC neurons Boston Childrens Hospital / Imperial College London CDKL5 $200,000 2016
Sila Ultanir, PhD Development of biomarkers for CDKL5 activity The Francis Crick Institute CDKL5 $150,000 2017
J. Silvio Gutkind, PhD Development of next-gen animal models for fibrous dysplasia (FD)/McCune-Albright syndrome (MAS) University California San Diego, Moores Cancer Center MDBR - Fibrous Dysplasia $58,500 2015
Igor Nestrasil, MD Discovery of brain MRI signatures in infants with severe form of MPS I in the pre-HSCT and post-HSCT stage University of Minnesota MPS I $330,543 2017
Maurizio Pacifici, PhD Drug Therapy for Hereditary Multiple Exostoses Children's Hospital of Philadelphia HTSC $109,548 2015
Li Changhong, MD, PhD Drug development for treatment of glutamate dehydrogenase hyperinsulinism Children's Hospital of Philadelphia MDBR - CHI $82,000 2016
Ashley Bush, MB BS, DPM, FRANZCP, PhD, FTSE Dual targeting of defective lipid and metal pathways in Niemann-Pick type C disease: an in vitro pilot study The Florey Institute of Neuroscience and Mental Health MDBR - NPC $50,000 2014
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