Research Involving:
Rare



Enhancing Treatment Response in Recurrent/Metastatic Osteosarcoma with Hydroxychloroquine

Enhancing Treatment Response in Recurrent/Metastatic Osteosarcoma with Hydroxychloroquine Principal Investigator: Dr. Nancy Gordon​ Disease: Osteosarcoma Research Description: Osteosarcoma (OS) is an orphan disease affecting approximately 400 children and adolescents...



Repurposing the Antiviral Drug Valganciclovir to Treat Cytomegalovirus-Induced Hearing Loss

Repurposing the Antiviral Drug Valganciclovir to Treat Cytomegalovirus-Induced Hearing Loss Principal Investigator: Dr. Joseph Rower​​ Disease: Hearing loss Research Description: Almost 400 children die each year from congenital cytomegalovirus (cCMV) infections, and...


Improving Outcomes for the Rare Liver Disease Biliary Atresia with a Cancer Drug in Vietnam

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...