Past awardees
“Piloting splice-modulating therapeutics for neurodegenerative diseases of childhood” – Timothy Yu, MD, PhD
“Development and validation of a protein-based urinary diagnostic assay for pneumococcal pneumonia (PP)” – Richard Malley, MD
“Translational potential of choroid plexus – autotaxin therapy for white matter injury in neonates” – Maria Lehtinen, PhD
“The Role of Disease Activity in Driving the Development of Lung Disease in Patients with sJIA” – Lauren Henderson, MD, MMSc
“Engineering hematopoietic stem cells for gene therapy without chemotherapy” – Daniel Bauer, MD, PhD
“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