Funded Awards

Funded Grants September 2021

PI Agency Title Megan Cooper NIH/ UG3/UH3/Baylor College of Medicine Safety and Efficacy of Itacitinib in treatment of JAK/STAT gain of function disorders Amjad Horani CDI Mechanisms of Primary Ciliary Dyskinesia Cynthia Ortinau Brain & Behavior Research Foundation Association of Parental Psychological Distress with Brain and Neurodevelopment in Congenital Heart …

Funded Awards

Funded Grants August 2021

PI Agency Title Fahd Ahmad NIH/CCHMC EMS for Children: Pediatric Emergency Care Applied Research Network (PECARN)  Jonathan Cooper NIH/R56 Characterizing and testing the efficacy of AAV-mediated gene therapy in a novel CRISPR/Cas9 generated sheepmodel of Cln1 disease. Jorge Di Paola NIH/R01/Ohio State University A Reversible Aptamer Based Therapeutic to Treat …

Funded Awards

Funded Grants July 2021

PI Agency Title Mohamed Abdelbaki EIF/The Hospital for Sick Children 3CI Study: Childhood Cancer Combination Immunotherapy. Phase 1b and Expansion Study of Nivolumab Combination Immunotherapy in Children, Adolescent and Young Adult (CAYA) Patients with Relapsed/Refractory Hypermutant Cancers Amy Armstrong Aflac Foundation/Children’s Healthcare of Atlanta A Maintenance Protocol of Sirolimus in …

Funded Awards

Funded Grants June 2021

PI Agency Title Mohamed Abdelbaki SLCH Foundation Pediatric Neuro-Oncology Fellowship 2021-2022 Francis Cole NIH Admin Supplement: Washington University School of Medicine Undiagnosed Diseases Network Clinical Site Megan Cooper SLCH Foundation Center for Pediatric Immunology Patricia Dickson NIH/R21 Gene therapy with modified GlcNAc-1-phosphotransferase for mucolipidosis Patricia Dickson NIH/T32 Postdoctoral Training Program …

Funded Awards

Funded Awards May 2021

PI Agency Title Mohamed Abdelbaki LilaBean Foundation/University of California, San Francisco A Phase I Trial Evaluating the Combination of Trametinib and Everolimus in Pediatric and Young Adult Patients with Recurrent Low Grade Gliomas Vikas Dharnidharka NIH/R01/The Children’s Mercy Hospital Reaching Equity for Adults and CHildren in Transplant (REACH-TRANSPLANT) Melanie Fields …

Funded Awards

Funded Awards February 2021

PI Agency Title Angela Brown NIH/U01 17/22 Diabetes Prevention Program Outcomes Study (DPPOS) Phase 3 – Research Project Juliane Bubeck Wardenburg NIH/R01 Development of human adaptive immunity to Staphylococcus aureus Abby Green DOD Mutagenic Deaminase Activity in Cancer Jeffrey Magee Leukemia & Lymphoma Society/Career Development Program Neonatal origins of pediatric …

Funded Awards

Funded Awards January 2021

PI Agency Title  F.S. Cole CDC/Task Force for Global Health, Inc COVID-19 Electronic Health Data Initiative  Brian DeBosch Doris Duke Charitable Foundation Supplement-Modeling Human Hepatocytes to Translate Glucose-fasting Mimetics as Novel Therapies Against Non-alcoholic Fatty Liver Disease.  Jorge Di Paola NIH/R33/University of Colorado A systems biology approach to identifying the …

Funded Awards

Funded Awards April 2020

  PI Agency Title Leonard Bacharier NIH/ U2C/Duke University ECHO Coordinating Center Jeffrey Bednarski NIH/U54/Regents of University of California – San Francisco Primary Immune Deficiency Treatment Consortium Brian DeBosch AASLD Hepatic Arginase 2 as a Novel Fasting-induced Factor to Treat NAFLD and NASH Lori Holtz NIH/R01 Role of Environmental Exposures …

Funded Awards

Funded Awards March 2020

  PI Agency Title Jagruti Anadkat NIH/R01/University of Michigan Sleep-disordered Breathing in Infants with Myelomeningocele Aimee Baumann-Dudenhoeffer NIH/R03 Longitudinal Effects of Diet and the Gut Microbiome on Immunosuppressive Drug Metabolism Juliane Bubeck Wardenburg Washington University Center for Drug Discovery/ Matching Micro Grant LEAP: Single optimized novel formulation of alpha-toxin vaccine …

Funded Awards

Funded Awards January 2020

  PI Agency Title Jonathan Cooper ForeBatten Foundation Defining Peripheral and Enteric Nervous System disease in Juvenile Batten Disease Jonathan Cooper Abeona Neuropathologic Evaluation of the Efficacy of Intrathecal AAV9-Mediated Gene Therapy for Cln1 Disease Jorge Di Paola NIH/R01 Genomics of Megakaryocyte and Platelet Biology Andrea Granados CDTR Safety and …