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 11 - 20 of 168

Awardee Project Title Institution Grant Amount Funding Year
Steven Rowe, MD, MPSH An open label N of 1 study to evaluate the safety and efficacy of long-term treatment with Ivacaftor in combination with ATALUREN (PTC124) in Subjects with Nonsense Mutation Cystic Fibrosis University of Alabama at Birmingham MDBR - Cystic Fibrosis 1282x $51,500 2015
Norbert Perrimon, PhD An shRNA based screening method for TSC synthetic lethal screens Harvard Medical School MDBR - LAM $50,500 2015
Charles Vite, DVM, PhD, BS Antemortem 3D EPSI metabolic mapping of the canine MPS I brain University of Pennsylvania MPS I $150,000 2013
Philip Stork, MD Are Raf dimers targets for therapeutic intervention in RASopathies? Oregon Health & Sciences University MDBR - RASopathies $44,000 2016
Dolan Sondhi, PhD Assessment of three routes of delivery of the IDUA gene (gene implicated in MPS-I) in non-human primates comparing two serotypes, AAV9 and AAVrh.10 for the treatment of MPS-I. Cornell University MPS I $225,000 2014
Jennifer Kalish, MD, PhD Beckwith-Wiedemann Syndrome Program of Excellence Feasibility Pilot Grant Children's Hospital of Philadelphia POE - Beckwith-Wiedemann Syndrome $60,000 2017
Diva De Leon-Crutchlow, MD, MSCE Bihormonal Bionic Pancreas for the Treatment of Diabetes Post-Pancreatectomy in Individuals with Congenital Hyperinsulinism – A Pilot Study University of Pennsylvania/Children's Hospital of Philadelphia MDBR - Congenital Hyperinsulinism $87,109 2018
Florian Eichler, MD Biodistribution of Intrathecal AAV9-mediated Gene Therapy in AMN Massachusetts General Hospital MDBR - ALD $100,000 2016
Igor Nestrasil, MD, PhD Brain MRI signatures in infants with infantile forms of GM-1 and GM-2 University of Minnesota MDBR - Tay-Sachs, Sandhoff, GM-1, or Canavan Disease $42,419 2018
Bianca De Filippis, PhD Brain Rho GTPases: in search of innovative therapeutic targets for CDKL5 deficiency Istituto Superiore di Sanità CDKL5 $150,000 2016
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