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 21 - 30 of 168

Awardee Project Title Institution Grant Amount Funding Year
Alysson Muotri, PhD CDKL5 Syndrome cortical organoids for drug testing and reversibility potential University of California San Diego CDKL5 $150,000 2017
Ljubica Caldovic, PhD CNS Protection from Hyperammonemia Children's National Health System MDBR - UCD $43,000 2015
Brittney Gurda, PhD CNS gene therapy to restore NPC1 protein expression in cerebellar Purkinje cells and delay or prevent disease onset in feline Niemann-Pick type C1 (NPC1) disease. University of Pennsylvania, School of Veterinary Medicine MDBR - NPC $35,000 2014
Alessandra Recchia, PhD CRISPR/Cas9 nuclease design to target allele-specific Collagen VI mutations in patients' fibroblasts University of Modena and Reggio Emilia MDBR - Congenital Muscular Dystrophy $41,831 2018
Xuntian Jiang, PhD Carboxyl lysosphingomyelin as a Biomarker for Diagnosis and Assessment of Treatment of NPC Disease Washington University School of Medicine MDBR - NPC $51,000 2016
Andrew Lieberman, MD Characterization of NPC1-I1061T proteostasis machinery for development of NPC therapeutics University of Michigan Medical School MDBR - NPC $53,500 2015
Magi Casal, Dr med vet, PhD, Dipl. ECAR Cognitive Studies in Dogs Affected with Mucopolysaccharidosis Type I (MPS I) After Intrathecal (IT) Therapy with AAV Vectors University of Pennsylvania MPS I $165,000 2014
Yair Anikster, MD, PhD Correction of splice mutations in Ataxia-Telangiectasia through novel antisense oligonucleotides Edmond and Lily Safra Children's Hospital - Sheba Medical Center MDBR - Ataxia-Telangiectasia $65,689 2018
Hasan Orhan Akman, PhD Could restoring GBE in affected tissues be a treatment for APBD? Columbia University Medical Center MDBR - Adult Polyglucosan Body Disorder $52,978 2018
Maria Kontaridis, PhD Delineating the cause of gastrointestinal abnormalities in RASopathy disorders using human inducible pluripotent stem cells (iPSCs) Beth Israel Deaconess Medical Center/Harvard Medical School MDBR - RASopathies $65,000 2014
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