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 121 - 130 of 168

Awardee Project Title Institution Grant Amount Funding Year
Albert Misko, MD, PhD Quantitative assessment of visual engagement in mucolipidosis type IV Massachusetts General Hospital MDBR - Mucolipidosis $71,939 2018
Annette Bley, PhD Quantitative description of the clinical course of Canavan disease University Medical Center Hamburg-Eppendorf, Dept of Pediatrics MDBR - Tay-Sachs $35,000 2014
Christopher Stephen, MB, CHB Quantitative oculomotor assessment in Late-Onset Tay-Sachs Massachusetts General Hospital, Harvard Medical School MDBR - Tay-Sachs $42,000 2016
Barry Cooperman, PhD Rational Design of Effective Therapeutics for Treatment of the 1282X CFTR Disorder University of Pennsylvania MDBR - CF1282x $53,000 2016
Tonis Timmusk Regulation of TCF4 transcriptional activity in neurons Tallinn University of Technology MDBR - Pitt Hopkins $50,000 2015
Amanda Ackerman, MD, PhD Repair of Gene Defects in Beta-Cells from Children with Congenital Hyperinsulinism Children's Hospital of Philadelphia Genome Editing $110,000 2016
Antoine Muchir Rescue of cardiac nicotinamide adenine dinucleotide (NAD+) content improves cardiac function in dilated cardiomyopathy in CMD caused by lamin A/C gene Center of Research in Myology UPMC MDBR - CMD $47,000 2016
Tommaso Pizzorusso, PhD Rescuing CDKL5 mice phenotype by targeting developmental critical period mechanisms Institute of Neuroscience, CNR CDKL5 $150,000 2017
Denise Adams, MD Retrospective analysis of the safety and efficacy of lymphatic anomalies of the bone (Generalized Lymphatic anomaly (GLA), Gorham Stout Disease (GSD), Channel type Lymphatic Malformation (CTLM)) treated with mTOR inhibition and bisphosphonates Boston Children's Hospital MDBR - Generalized Lymphatic Anomaly (lymphangiomatosis) & Gorham-Stout Disease $51,109 2018
Yogesh Chutake, PhD Reversal of epigenetic promoter silencing in Friedreich ataxia by histone deacetylase inhibitors University of Oklahoma HSC MDBR - FA $35,000 2014
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