Using a Cancer Drug to Treat a Rare Pediatric Liver Disease

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...

Repurposing an Antibiotic to Treat a Defect in Vitamin D Metabolism

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...