Research and News Involving:
Rare Disease
Adult, CureAccelerator Live!, Dr. Phillips, Drug, Minority/Underserved, 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 (ALL) 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, 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, 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, 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, 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, Neuro, 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...
CureAccelerator Live!, Current Research, 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...
Adult, Current Research, Dr. Zacks, Drug, Ophthalmic, Rare Disease, University of Michigan
Testing a Generic Malarial Drug in a Rare Ophthalmic Condition, Retinitis Pigmentosa Principal Investigator: Dr. David Zacks Disease: Retinitis Pigmentosa Research Description: Certain mutations are a common cause of retinitis pigmentosa (RP), a rare ophthalmic...
Adult, CureAccelerator Live!, Current Research, Dr. Swiecicki, Drug, Longest Day of Golf, Oncology, Rare Disease, University of Michigan
Repurposing Old Drugs as New Therapies for Metastatic Thyroid Cancer Principal Investigator: Dr. Paul Swiecicki Disease: Thyroid Cancer Research Description: Thyroid cancer is the sixteenth most common malignancy in the world. Although most patients are cured with a...
Adult, Dr. Pettit, Drug, Oncology, Rare Disease, University of Michigan
Using a Skin Cancer Drug to Improve Current Treatment in a Rare Blood Cancer, Myelofibrosis Principal Investigator: Dr. Kristen Pettit Disease: Myelofibrosis Research Description: Myelofibrosis (MF) is a rare, aggressive bone marrow cancer causing excessive scar...
Adult, Combo, Completed Research, Dr. Halatsch, Drug, Neuro, Oncology, Rare Disease, Ulm University
Combining Nine Repurposed Drugs with a Current Chemotherapy Treatment in Adult Brain Cancer Principal Investigator: Dr. Marc-Eric Halatsch Disease: Glioblastoma Research Description: For decades, new experimental therapies have repeatedly failed to improve the...
Adult, Completed Research, CureAccelerator Live!, Dr. Arrillaga-Romany, Dr. Kouwenhoven, Dr. Tannous, Dr. Van Linde, Drug, Harvard Medical School, Longest Day of Golf, Massachusetts General Hospital, Neuro, Oncology, Rare Disease, VUmc Cancer Center Amsterdam
A Novel Combination of Generic Chemotherapy Drugs to Treat Brain Cancer Principal Investigators: Dr. Bakhos Tannous, Dr. Isabel Arrillaga-Romany, Dr. Mathilde Kouwenhoven, Dr. Myra Van Linde Disease: Glioblastoma Research Description: Glioblastomas (GBMs) are...
Adult, Completed Research, Dr. Furr-Stimming, Drug, McGovern Medical School at The University of Texas Health Science Center at Houston, Neuro, Rare Disease
Treating Irritability in Huntington’s Disease with a Repurposed Neurological Drug Principal Investigator: Erin Furr-Stimming, MD Disease: Huntington’s Disease Research Description: Huntington’s disease (HD) is a devastating inherited neurologic disease that...
Adult, Completed Research, Dr. Anderson, Drug, Georgetown University, Mid-Atlantic, Neuro, Rare Disease, Uncategorized
Using a Cancer Drug in Huntington’s Disease Principal Investigator: Dr. Karen Anderson Disease: Huntington’s Disease Research Description: Huntington’s disease (HD) is a rare, inherited brain disease, which causes uncontrollable, fast movements, memory and...
