Big Leap Research, Current Research, Developing World, Dr. Nguyen, Drug, Minority/Underserved, Pediatric, Rare, 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...
Chicago, CureAccelerator Live!, Current Research, Dr. Lieber, Drug, Neuro, Pediatric
Repurposing a Blood Cancer Treatment to Treat Muscle Contractures in Cerebral Palsy Patients Principal Investigator: Dr. Richard Lieber Disease: Cerebral palsy Research Description: Cerebral palsy (CP) is the most common cause of childhood motor disability, affecting...
Children's Hospital of Philadelphia, Current Research, Diagnostic, Dr. Seif, Drug, Mid-Atlantic, Oncology, Pediatric, Rare
Preventing Relapse After Bone Marrow Transplant in Pediatric Acute Lymphoblastic Leukemia (ALL) with a Personalized Treatment Principal Investigator: Dr. Alix Seif Disease: Acute Lymphoblastic Leukemia Research Description: Pediatric acute lymphoblastic leukemia (ALL)...
Current Research, Developing World, Dr. Chang, Drug, George Washington University, Infectious Disease, Mid-Atlantic, Minority/Underserved, 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, Longest Day of Golf, Pediatric, Rare, Uncategorized, 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, Mid-Atlantic, Pediatric, Rare, 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...
