Current Environment:

Past awardees


“The Arch Watch Study: an integrated evaluation of hemodynamics during monitoring of ductus arteriosus closure in infants with suspected coarctation of the aorta” – John Kheir, MD

“The role of IL-17 cytokine family in Kawasaki Disease” – Pui Lee, MD, PhD

“Antisense oligonucleotide therapy for calmodulinopathy” – William Pu, MD, PhD


“The Mechanism of Neurocognitive Dysfunction in CPVT” - Vassilios Bezzerides, MD, PhD

“Antisense oligonucleotide therapy for calmodulinopathy” - William Pu, MD

“Developing astrocyte-directed SSADH restoration gene therapy” - Alexander Rotenberg, MD, PhD


“Correction of Erythropoietic Protoporphyria by Gene Editing Hematopoietic Stem Cells” - Mark Fleming, MD, DPhil

“Use of CDK6 Inhibitors to Treat Familial Platelet Disorder with Propensity to Develop Leukemia” - Alan Cantor, MD, PhD

“Coupled Electric and Magnetic Neuromodulation for Seizure Suppression” - Alexander Rotenberg, MD, PhD

“Targeting the fungal cell wall to develop potent new candidacidal agents” - Julia Koehler, MD

“Developing a PET-based method to assess myelination-promoting treatments” - Zhigang He, PhD, BM


“Development of a near universal curative gene editing therapy for Erythropoietic Protoporphyria” - Mark Fleming, MD, DPhil

“Computer modeling for prospective design of patches for aortic arch reconstruction” - David Hoganson, MD

“Gene Therapy via Genome Modification by Cas9 and AAV-Mediated Homology Directed Repair” - William Pu, MD


“Novel strategy for the generation of potent cancer vaccines that elicit CD4 T cell immunity” - Roberto Chiarle, MD

“Effects of Nutritional Ketosis on Brain Response to Hypoglycemia in People with Type 1 Diabetes Mellitus” - Belinda Lennerz, MD, PhD

“Repurposing disulfiram to treat pediatric autoinflammatory diseases” - Judy Lieberman, MD, PhD

“Gene Therapy for Diamond-Blackfan Anemia” - Vijay Sankaran, MD, PhD

“Development of a circulating tumor DNA assay for early cancer detection in children with Li-Fraumeni syndrome” - Brian Crompton, MD


“Studies in Early Life Epilepsy: KCNQ2-Mediated Hyperexcitability in iPSC-Derived Neurons” - Annapurna Poduri, MD, MPH

“Modeling dystrophic cardiomyopathy in vitro using iPSC and genome editing” - Da-Zhi Wang

“Targeting Telomerase via Non-coding RNA Pathways in Cancer” - Suneet Agarwal, MD, PhD

“Bowel-lengthening Robotic Implant That Can Be Removed Without Surgery” - Pierre Dupont, PhD

“Non-Sedated Feed and Wrap MRI and Deep Learning Tools for Automated Radiation-Free Kidney Function Quantification” - Sila Kurugol, PhD


“Development of circulating tumor DNA assays for pediatric sarcomas” - Brian Crompton, MD

“Robotic implant for mechanotransduction-based bowel lengthening” - Pierre Dupont, PhD

“Development of efficient gene-therapy approaches for delivery of large gene sequences to the inner ear” - Jeffrey Holt, PhD

“Characterization of alterations in premature infant cerebral blood flow in association with transfusions using continuous NIRS technology, a pilot study” - Kristen Leeman, MD

“A gene therapy approach for CPVT, a potentially lethal inherited arrhythmia” - William Pu, MD


“Poly (A) polymerase inhibitors for the treatment of telomere diseases” - Suneet Agarwal, MD, PhD

"Biology studies of hematopoietic stem and progenitor cells mobilized by plerixafor from patients with sickle cell disease: - Daniel Bauer, MD, PhD

“Development of CAR T cells against the ALK oncogene” - Roberto Chiarle, MD

“Development of Circulating Tumor DNA Assays for EwingSarcomas” - Brian Crompton, MD

“A Therapeutic Strategy for Atopic Dermatitis by Targeting the WNT Pathway” - Xi He, PhD

“Human trabecular bone endothelial cells regulate osteoregeneration” - Juan Melero-Martin, PhD

“Development of a gene therapy model for DOCK8 deficiency” - Sung-Yun Pai

“Microbiome and Metabolomic Changes in Exhaled Breath Condensate as a Biomarker of Extraesophageal Reflux Disease” - Rachel Rosen, MD, MPH


"A Biometric Direct Cardiac Compression Device for Ischemic Cardiomyopathy” - Frank Pigula, MD

“Cdk4/6 inhibition for T-cell Acute Lymphoblastic Leukemia Therapy” - Yana Pikman, MD

“The role of Fecal Microbiota Transplant in causing sustained unresponsiveness after peanut oral immunotherapy in peanut allergy patients” - Rima Rachid, MD

“Measuring oxidative stress and its impact on cortical inhibition by MRS and ppTMS following traumatic brain injury” - Alexander Rotenberg, MD, PhD

“Toward in vivo neuroimaging biomarkers of concussion: evaluation with a mouse model of repetitive mild traumatic brain injury (rmTBI)” - Benoit Scherrer, PhD


“Predicting Regression and Response to Treatment in Rett Syndrome” - Michela Fagiolini, PhD

“Identification of candidate Staphylococcus aureus vaccine antigens by RNA expression profile analysis of human abscesses” - Kristin Moffitt, MD

“A Biometric Direct Cardiac Compression Device” - Frank Pigula, MD

“Serum Biomarkers Predict Need for Erythropoietin Treatment in Preterm Infants” - Shenandoah Robinson, MD

“Urine Proteomics to Identify Biomarkers for Tuberculosis” - Hanno Steen, PhD


“Cellular and Molecular Basis of Lymphatic Malformations” - Joyce Bischoff, PhD

“Regulatory T Cell Bio-Markers of Food Allergy Outcomes” - Talal Chatila, MD

“ALK Tumor Vaccine in Neuroblastoma” - Roberto Chiarle, MD

“Novel Cardiac Biomarkers and Tissue Doppler Imaging for Rejection Surveillance after Pediatric Heart Transplantation” - Steven Colan, MD, FACC, FASE

“Novel Strategies for Intracardiac Septal Defect Closure” - Pedro del Nido, MD

“Pre-Clinical and Clinical Testing of NMDAr Antagonist Treatment in Rett Syndrome” - Michela Fagiolini, PhD

“Accelerated 3D-SSFP Magnetic Resonance Imaging for Guiding Congenital Heart Disease Management” - Mehdi Hedjazi Moghari


“Biomarkers for Early Identification of Children with Epileptic Encephalopathies” - William Bosl, PhD

“The Effects of Inosine in Promoting Recovery after Spinal Cord Injury” - Larry Benowitz

“A Novel Light-Activated Adhesive for Pediatric Cardiovascular Applications” - Pedro del Nido, MD 

“Role of B7.1 in Diabetic Nephropathy: Establishing the CTLA4-Ig Clinical Trial” - Paolo Fiorina, MD, PhD

“A Novel Strategy for Turning off b-catenin Oncogenic Signaling in Cancer” - Xi He, PhD

“Pilot trial of Glycine Supplementation in Autosomal Recessive Congenital Sideroblastic Anemia due to Mutations in SLC25A38” - Matthew Heeney, MD

“Early Validation of Urinary Biomarkers of Renal Obstruction” - Richard Lee, MD

“Development of a Multiple Antigen Presentation System (MAPS) Pneumococcal Vaccine” - Richard Malley, MD

“Regeneration of the Anterior Cruciate Ligament after Rupture” - Martha Murray, MD

“Safe Food Allergy Desensitization using IgE Receptor Signaling Inhibitors” - Hans Oettgen, MD, PhD

“Diffusion-weighted MRI for the Assessment of Disease Activity and Progression in Pediatric Chron’s Disease” - Simon Warfield, PhD


“Role of B7.1 in Diabetic Nephropathy” - Paolo Fiorina, MD, PhD

“Validation of a genetically engineered mouse model of neuroblastoma drive by mutant ALK and MYCN” - Rani George, MD, PhD

“Recovery from Amblyopia with Cholinesterase Inhibitors” - Takao Hensch, PhD

“New Genetic Classes of Idiopathic Short Stature Defined by Sequence and Copy Number Variants” - Joel Hirschhorn, MD, PhD

“Stress-potentiated screening to identify novel inhibitors of M. tuberculosis” - Robert Husson, MD; Walter Kaufmann, MD

“Potential Clinical Biomarkers of SIDS” - Hannah Kinney, MD

“Development of Contrast Enhanced Quantitative CT as Clinical Tool for Arthritis” - Brian Snyder, MD, PhD


“Effects of a food preservative on glucose Homeostasis” - David Ludwig, MD, PhD

“c-Met inhibitor treatment of Polycystic Kidney Disease” - Jordan Kreidberg, MD, PhD

“Deciphering autism through cross-disease system biology” - Dennis Wall, PhD

“A novel therapy for severe food allergy by rapid and safe induction of oral tolerance” - Dale Umetsu, MD, PhD

“Reducing ventilator-associated lung injury through non-invasive bedside imaging” - Gerhard Wolf, MD

“Closed-loop adjustment of transcranial direct current stimulation (tDCS) in of status epilepticus” - Alexander Rotenberg, MD, PhD

“Chemical inhibition of a novel Wnt modulator for osteoporosis therapeutics” - Xi He, PhD


“Use of engineered meganucleases to correct RAG1 deficiency in humans” - Luigi Notarangelo, MD

“A novel therapy for severe food allergy by rapid and safe induction of oral tolerance” - Dale Umetsu, MD, PhD

“Novel platform for mucosal protein drug/vaccine delivery and increased half-life” - Wayne Lencer, MD

“Treatment of Status Epilepticus by Transcranial Direct Current Stimulation” - Alexander Rotenberg, MD, PhD

“A stem/progenitor cell that recapitulates infantile hemangioma” - Joyce Bischoff, PhD

“Pilot Trial of Bumetanide to Treat Neonatal Seizures” - Janet Soul, MDCM

“Urine Proteomics of Kawasaki Disease and Pediatric Vasculitis” - Hanno Steen, PhD


“Proteome-wide screen for M. tuberculosis antigens” - Robert Husson, MD

“A transcutaneous vaccine to prevent pneumococcal colonization and disease” - Richard Malley, MD

“GDNF receptor agonists to treat peripheral neuropathies” - Gabriel Corfas, PhD

“A stem/progenitor cell that recapitulates infantile hemangoma” - Joyce Bischoff, PhD

“The essentiality of archidonic acid (AA), docosahexaaenoic acid (DHA)” - Mark Puder, MD, PhD

“Role of ER Stress in Development of Lepin Resistance” - Umut Ozcan, MD

“Microparticles with TRPV4 inhibitor for lung protection” - Benjamin Matthews, MD

“Reversal of Glucocorticoid Resistance in Acute Lymphoblastic Leukemia” - Scott Armstrong, MD, PhD

“Novel Approaches in Targeting Pediatric B-ALL” - Leslie Silberstein, MD