Research and News Involving:
Pediatric
Big Leap Research, Current Research, Dr. Nguyen, Drug, Health Equity, Liver Disease, LMICs, Pediatric, Rare Disease, ReGRoW, Vietnam National Children’s Hospital
Improving Outcomes for the Rare Liver Disease Biliary Atresia with a Cancer Drug in Vietnam Principal Investigator: Dr. Anh-Hoa Pham Nguyen Disease: Biliary atresia Research Description: Biliary atresia (BA) is a rare congenital liver disease that causes bile duct...
Children's Hospital of Philadelphia, Current Research, Diagnostic, Dr. Seif, Drug, Mid-Atlantic, Oncology, Pediatric, Rare Disease
Preventing Relapse After Bone Marrow Transplant in Pediatric Acute Lymphoblastic Leukemia with a Personalized Treatment Principal Investigator: Dr. Alix Seif Disease: Acute Lymphoblastic Leukemia Research Description: Pediatric acute lymphoblastic leukemia (ALL)...
Current Research, Dr. Chang, Drug, George Washington University, Health Equity, Infectious Disease, LMICs, Mid-Atlantic, Pediatric
Repurposing a Flu Treatment for Severe Dengue Patients in Colombia Principal Investigator: Dr. Aileen Chang Disease: Dengue Fever Research Description:Dengue viruses (DenV) are among the leading causes of pediatric morbidity and mortality globally, with 96 million...
Chicago, Children's National Health System, Completed Research, Dr. Holterman, Dr. Nadler, Drug, Liver Disease, Longest Day of Golf, Pediatric, Rare Disease, University of Illinois at Chicago
Using a Cancer Drug to Treat a Rare Pediatric Liver Disease Principal Investigator: Dr. Evan Nadler, Dr. Ai-Xuan Holterman Disease: Biliary Atresia Research Description: Biliary atresia (BA) is a rare disease, occurring in approximately 1 in 10,000-20,000 live...
Children's Hospital of Philadelphia, Completed Research, Dr. Levine, Dr. Sochett, Drug, Metabolic Disorder, Mid-Atlantic, Pediatric, Rare Disease, The Hospital for Sick Children
Repurposing an Antibiotic to Treat a Defect in Vitamin D Metabolism Principal Investigator: Dr. Michael Levine, Dr. Étienne Sochett Disease: CYP24A1 mutations, hypercalcemia, hypercalciuria Research Description: Patients with loss of function mutations in the CYP24A1...
Completed Research, Dr. Riggins, Drug, Johns Hopkins Medicine, Mid-Atlantic, Oncology, Pediatric, Rare Disease
A Phase I Trial for Recurrent Pediatric Brain Cancers Using a Repurposed Generic Drug Principal Investigator: Dr. Gregory Riggins Disease: Medulloblastoma, Glioblastoma Research Description: Medulloblastoma is the most common malignant brain tumor in children, and...
Completed Research, Dr. Leary, Drug, Oncology, Pediatric, Rare Disease, Seattle Children's
Repurposing Drugs for Recurrent Pediatric Brain Tumors Principal Investigator: Dr. Sarah Leary Disease: Pediatric Brain Cancer Research Description: Pediatric brain tumors are the most common solid tumor malignancy in children, and continue to be a leading cause of...
Completed Research, Dr. Crystal, Neurology, Other, Pediatric, Rare Disease, Weill Cornell Medical College
Repurposing Gene Therapy for Batten Disease Principal Investigator: Dr. Ron Crystal Disease: Batten Disease Research Description: Batten Diseases comprise a group of genetic diseases that affect children. Children with these gene defects are missing key enzymes that...
Completed Research, Dr. Hollinger, Drug, Johns Hopkins Medicine, Mid-Atlantic, Neurology, Pediatric
Repurposing a Generic Drug in the Treatment of Pediatric Depression Principal Investigator: Dr. Kristen Hollinger Disease: Pediatric Depression Research Description: Suicide due to major depressive disorder (MDD) has simultaneously become a leading cause of death...
Cincinnati Children's Hospital Medical Center, Completed Research, Dr. Wink, Drug, Longest Day of Golf, Neurology, Pediatric
Repurposing a Generic Drug in Autism Spectrum Disorder Principal Investigator: Dr. Logan Wink Disease: Autism Research Description: One in 68 children is diagnosed with an Autism Spectrum Disorder (ASD). Individuals with ASD endure lifelong deficits in social...
Completed Research, CureAccelerator Live!, Dr. Nichols, Drug, Immune Disorder, Longest Day of Golf, Pediatric, Rare Disease, St. Jude Children's Research Hospital
Repurposing a Blood Cancer Drug to Treat an Immune Disorder (HLH) in Children Principal Investigator: Dr. Kim Nichols Disease: Hemophagocytic Lymphohistiocytosis Research Description: Hemophagocytic lymphohistiocytosis (HLH) is a rare, poorly understood disorder of...
Dr. Bönnemann, Dr. Dowling, Drug, Muscular Disorder, National Institutes of Health, Pediatric, Rare Disease, The Hospital for Sick Children
Repurposing a Cancer Drug to Treat a Rare Disease Principal Investigator: Dr. James Dowling and Dr. Carsten Bönnemann Disease: Myotubular Myopathy Research Description: X-linked myotubular myopathy (MTM) is a rare disorder that affects males and is caused by mutations...
Completed Research, CureAccelerator Live!, Dr. Budree, Dr. Osman, GI, Health Equity, LMICs, Longest Day of Golf, Openbiome, Other, Pediatric
The THRIVE Study: Repurposing Fecal Microbiota Transplantation for the Treatment of Pediatric Malnutrition in South Africa Principal Investigator: Dr. Majdi Osman, Dr. Shrish Budree Disease: Severe and Moderate Pediatric Malnutrition Research Description: Globally,...
Boston Children's Hospital, Completed Research, CureAccelerator Live!, Dr. Kahn, GI, Infectious Disease, Other, Pediatric
Fecal Microbiota Transplantation Patient Registry for Pediatric C-diff Principal Investigator: Dr. Stacy Kahn Disease: Clostridium difficile infection Research Description: Fecal microbiota transplantation (FMT) is the repurposing of human stool to deliver healthy...
