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 CDKL5 Program of Excellence Awardees

Awarded Grants

Displaying Results 131 - 140 of 175

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
Stephen Stick, MB, Bchir, PhD, MRCHP, FRACP Epithelial-mesenchymal transition in paediatric airway epithelial cells following infection with bronchiolitis obliterans associated adenoviruses. Telethon Kids Institute MDBR - Bronchiolitis Obliterans $50,500 2015
Garry Cutting, MD Systematic Evaluation of a Cluster of Nonsense Mutations in Exon 22 of CFTR to Inform Treatment Johns Hopkins University School of Medicine MDBR - Cystic Fibrosis $69,000 2015
Joseph Rutkowski, PhD Inducible lymphatic hyperplasia to drive chylous accumulation mimicking lymphangiomatosis Texas A&M Health Science Center MDBR - GLA/GSD $51,000 2015
Igor Nestrasil, MD Microstructural and functional MRI signatures in patients with MPS I University of Minnesota MDBR - MPS $50,500 2015
Andrea Ballabio, MD Modulation Of Cellular Clearance To Treat Mucopolysaccharidoses Type 1 The Telethon Institute of Genetics and Medicine MPS I $300,000 2014
Michael James, Dphil Structure-guided development of small molecule “pharmacological chaperones” for the treatment of Mucopolysaccharidosis I University of Alberta MPS I $300,000 2014
Lorne Clarke, MD Developing Substrate Reduction Therapy for MPS I, II & III University of British Columbia MPS I $140,650 2014
Wei Zheng. PhD Phenotypic screening to identify lead compounds that reduce lysosomal storage in neuronal cells derived from MPS I patients National Institutes of Health MPS I $150,000 2014
Jude Samulski, PhD Gene Therapy for MPS Ocular Blindness in BMT Individuals University of North Carolina School of Medicine MPS I $330,000 2014
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