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)...
Adult, Current Research, Dr. Park, Ophthalmic, Other, Rare, University of California Davis
Repurposing Bone Marrow Stem Cell Therapy for Vision Loss Caused by Retinitis Pigmentosa Principal Investigator: Dr. Susanna Park Disease: Retinitis Pigmentosa Research Description: Retinitis pigmentosa (RP) is a rare hereditary form of retinal degeneration that can...
Adult, CureAccelerator Live!, Current Research, Nutraceutical, Oncology, Rare, University of California Irvine
Treating a Group of Rare Blood Cancers, Myeloproliferative Neoplasms, with a Nutraceutical Principal Investigator: Dr. Angela Fleischman Disease: Myeloproliferative neoplasms: Polycythemia vera, essential thrombocythemia and myelofibrosis Research Description:...
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...
Completed Research, Dr. Riggins, Drug, Johns Hopkins Medicine, Mid-Atlantic, Oncology, Pediatric, Rare
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...
