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 …

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 …

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 …