Research and News Involving:
Rare Disease
Adult, Completed Research, Dr. Ntekim, Drug, Health Equity, LMICs, Oncology, Rare Disease, ReGRoW, University College Hospital Ibadan, Women's Health
Adding the Antibiotic Clofazimine to Chemotherapy for Triple Negative Breast Cancer in Nigeria Principal Investigator: Dr. Atara Ntekim Disease: Breast cancer Research Description: Breast cancer causes high cancer related death globally. A type called triple negative...
Adult, Cincinnati Children's Hospital Medical Center, Current Research, Dr. Abonia, Drug, Health Equity, Immune Disorder, Rare Disease
Repurposing a Rare Disease Treatment for the Immune Disorder Eosinophilic Esophagitis Principal Investigator: Dr. J. Pablo Abonia Disease: Eosinophilic esophagitis Research Description: Eosinophilic esophagitis (EoE) is a rare disease caused when the immune system...
Completed Research, CureAccelerator Live!, Dr. Gordon, Drug, Health Equity, Oncology, Pediatric, Rare Disease, The University of Texas MD Anderson Cancer Center
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...
Adult, Chicago, CureAccelerator Live!, Current Research, Drug, Loyola University Medical Center, Oncology, Rare Disease, Women's Health
Repurposing the Blood Pressure Drug Minoxidil for the Treatment of Recurrent Chemo-Resistant Ovarian and Other Gynecological Cancers Principal Investigator: Dr. Margaret Liotta Disease: Ovarian cancer Research Description: Epithelial ovarian cancer is the most lethal...
Completed Research, CureAccelerator Live!, Dr. Teng, Drug, Pediatric, Rare Disease, Stanford University, Vascular
Treating Vascular Malformations Using the Cancer Drug Trametinib Principal Investigator: Dr. Joyce Teng Disease: Extracranial arteriovenous malformations Research Description: Extracranial arteriovenous malformation (AVM) is a network of abnormal blood vessels more...
Completed Research, CureAccelerator Live!, Dr. Rower, Drug, Ear Nose Throat, Hearing Loss, Infectious Disease, Inner Ear, Pediatric, Rare Disease, University of Utah
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...
Combo, Drug, Oncology, Pre-clinical, Prof. Allen, Rare Disease, University of Cambridge
Testing two generic drugs to improve outcomes in osteosarcoma, a rare bone cancer Principal Investigator: Prof. Matthew Allen Disease: Osteosarcoma Research Description: Although osteosarcoma (OSA) is a rare type of cancer, it is the most common bone cancer in...
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...
Adult, CureAccelerator Live!, Dr. Phillips, Drug, Health Equity, Oncology, Rare Disease, University of Michigan
Improving Patient Response in Mantle Cell Lymphoma with Venetoclax Principal Investigator: Dr. Tycel Phillips Disease: Mantle cell lymphoma Research Description: Mantle cell lymphoma (MCL) is a rare form of non-Hodgkin lymphoma (NHL) that is diagnosed mostly in older...
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)...
Adult, Completed Research, Dr. Park, Inherited Disorder, Ophthalmic, Other, Rare Disease, University of California, 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, Dr. Fleischman, Drug, Los Angeles, Oncology, Rare Disease, University of California, University of California Irvine
Treating a Group of Rare Blood Cancers, Myeloproliferative Neoplasms, with N-Acetylcysteine 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, 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...
Adult, Completed Research, Device, Dr. Davalos, Dr. Martin, Mid-Atlantic, Oncology, Rare Disease, Virginia Tech University
Using an Approved Device to Increase the Immune Response in Pancreatic Cancer Patients Principal Investigator: Dr. Rafael Davalos Co-Investigator: Dr. Robert C.G. Martin Disease: Pancreatic Cancer Research Description: Irreversible electroporation (IRE) is a...
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, 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...
