2017
- AN ENDOPLASMIC RETICULUM CALCIUM STABILIZER FOR THE TREATMENT OF WOLFRAM SYNDROME(website)
- AN ORAL ULTRA LONG-ACTING IVERMECTIN FOR MALARIA ELIMINATION(website)
- CONFERENCE ON CLINICAL RESEARCH FOR RARE DISEASES (CCRRD)(website)
- DRUG DEVELOPMENT FOR TUBEROUS SCLEROSIS COMPLEX AND OTHER PEDIATRIC EPILEPTOGENIC DISEASES USING NEUROVASCULAR AND CARDIAC MICROPHYSIOLOGICAL MODELS(website)
- ENHANCING PATIENT ENGAGEMENT IN NEUROFIBROMATOSIS CLINICAL TRIALS(website)
- GENE THERAPY PLATFORM FOR RARE DISEASES(website)
- IDENTIFICATION OF ACTIVATORS OF PMP22 EXPRESSION FOR TREATMENT OF CMT1A(website)
- IDENTIFICATION OF INHIBITORS OF ZIKA PROTEASE (NS2B-NS3)(website)
- IDENTIFICATION OF NOVEL ANTIMARIAL DRUGS TARGETING ASEXUAL BLOOD STAGE OF P. VIVAX(website)
- IDENTIFICATION OF SMALL MOLECULE ER MODULATORS FOR WOLFRAM SYNDROME(website)
- IN VIVO EDITING FOR HEMOPHILIA GENE THERAPY(website)
- JUVENILE MYOSITIS(website)
- METARRESTIN FOR THE TREATMENT OF PANCREATIC CANCER(website)
- NOVEL NANOPARTICLE-BASED ENZYME REPLACEMENT THERAPY FOR HUNTER SYNDROME(website)
- P97 INHIBITORS FOR TREATMENT OF IBMPFD/ALS(website)
- PERSONALIZED NEURAL STEM CELL THERAPY FOR CANCER(website)
- PRECLINICAL EVALUATION OF VORINOSTAT IN ALOPECIA AREATA(website)
- PRKACA FOR FIBROLAMELLAR HEPATOCELLULAR CARCINOMA(website)
- QHTS TO IDENTIFY COMPOUNDS AGAINST MERKEL CELL CARCINOMA (MCC)(website)
- QHTS TO IDENTIFY INHIBITORS OF ZIKA VIRUS(website)
- QUANTITATIVE SYSTEMS PHARMACOLOGY APPROACH TO IDENTIFY CYTOPROTECTIVE AGENTS AND PATHWAYS FOR HUNTINGTON’S DISEASE(website)
- RARE AND UNDIAGNOSED DISEASES: DISCOVERY AND MODELS OF PRECISION THERAPY(website)
- RECONSTITUTED HIGH DENSITY LIPOPROTEIN PARTICLES AS SIRNA CARRIERS(website)
- SOFTWARE PLATFORM TO STRATIFY PATIENTS FOR TREATMENT ARM RANDOMIZATION IN HUMAN CLINICAL TRIALS USING PATIENT-LEVEL PREDICTIVE MODELS(website)
- SUBCUTANEOUS REPLACEMENT ENZYMES FOR FABRY DISEASE(website)
- TARGETING GLUCOSE METABOLISM FOR THE TREATMENT OF HEPATOCELLULAR CARCINOMA(website)
- USING THE PREIMPLANTATION FACTOR (PIF) TO TREAT GRAFT-VERSUS-HOST DISEASE(website)
- UTILIZATION OF PHENOTYPIC PRECISION MEDICINE TO IDENTIFY OPTIMAL DRUG COMBINATIONS FOR THE TREATMENT OF HEPATOCELLULAR CARCINOMA(website)
2016
- 3D BIOPRINTING OF HUMAN NATIVE-LIKE TISSUES AS DISEASE-IN-A-DISH MODELS FOR DRUG DISCOVERY(website)
- A DRUG BASED APPROACH FOR INTEGRIN-MEDIATED ALLEVIATION FOR MUSCULAR DYSTROPHY(website)
- A DRUG-SCREENING PLATFORM FOR AUTISM SPECTRUM DISORDERS USING HUMAN NEURONS AND ASTROCYTES(website)
- A NOVEL COMPOUND FOR TARGETED TREATMENT OF CBF LEUKEMIA(website)
- A PROTEIN REPLACEMENT DRUG FOR FRIEDREICHS ATAXIA(website)
- ALPHA-1 ANTITRYPSIN (AAT) DEFICIENCY(website)
- BIOMEDICAL DATA TRANSLATOR TECHNICAL FEASIBILITY ASSESSMENT AND ARCHITECTURE DESIGN(website)
- CONFERENCE ON CLINICAL RESEARCH FOR RARE DISEASES (CCRRD)(website)
- CTSA SUPPLEMENT(website)
- CYCLODEXTRIN FOR NIEMANN-PICK TYPE C1 DISEASE(website)
- DEVELOPMENT OF BONE MORPHOGENETIC PROTEIN INHIBITORS TO TREAT BLOOD AND BONE DISORDERS(website)
- DEVELOPMENT OF MALARIA TRANSMISSION-BLOCKING DRUGS(website)
- DEVELOPMENT OF NEUROSTEROIDS FOR LYSOSOMAL STORAGE DISORDERS(website)
- DEVELOPMENT OF NOVEL ANTIMALARIAL TARGETS AND COMBINATION THERAPIES TO ADDRESS DRUG RESISTANCE(website)
- DEVELOPMENT OF NUCLEIC ACID DELIVERY PLATFORM BASED ON POLYMERIC CXCR4 ANTAGONISTS(website)
- DEVELOPMENT OF PLATELET TRANSCRIPTOME SIGNATURES IN PATIENTS WITH MYELOPROLIFERATIVE NEOPLASMS(website)
- DEX-M74 FOR GNE MYOPATHY(website)
- DISEASE-SPECIFIC INTEGRATED MICROPHYSIOLOGICAL HUMAN TISSUE MODELS(website)
- EARLY CHECK: A COLLABORATIVE INNOVATION TO FACILITATE PRE-SYMPTOMATIC CLINICAL TRIALS IN NEWBORNS(website)
- FOURTH INTERNATIONAL MEDICAL SYMPOSIUM ON ERDHEIM-CHESTER DISEASE(website)
- GLYCOGEN STORAGE DISEASE III ENGINEERED HUMAN SKELETAL MUSCLE RARE DISEASE MODEL(website)
- HIGH CONTENT SCREENING USING 3-D MODELS OF OVARIAN CANCER(website)
- HTS ENABLED EXAMINATION OF DRUG COMBINATIONS(website)
- HUMAN DNA TRANSLESION SYNTHESIS POLYMERASE KAPPA(website)
- IDENTIFICATION AND VALIDATION OF TARGETS FOR THERAPEUTIC INTERVENTION IN RARE DISEASES OF INTERMEDIARY METABOLISM(website)
- IDENTIFICATION OF GPR52 ANTAGONISTS AS POTENTIAL TREATMENTS FOR HUNTINGTON DISEASE(website)
- IDENTIFICATION OF INHIBITORS OF PLASMODIUM FALCIPARUM HEXOKINASE (PFHK)(website)
- IDENTIFICATION OF INHIBITORS OF THE N370S MUTANT FORM OF GLUCOCEREBROSIDASE AS A POTENTIAL THERAPY FOR GAUCHER DISEASE(website)
- IDENTIFICATION OF INHIBITORS OF YES1 KINASE ACTIVITY AND DETERMINATION OF YES1 POLYPHARMACOLOGY OF KINASE INHIBITORS(website)
- IDENTIFICATION OF SMALL MOLECULE INHIBITORS OF PHF5A FOR GLIOBLASTOMA(website)
- IDENTIFICATION OF SMALL MOLECULE THAT ACT ON GSP; THE ETIOLOGIC MUTATION RESPONSIBLE FOR FIBROUS DYSPLASIA/MCCUNE-ALBRIGHT SYNDROME(website)
- IDENTIFICATION OF SMALL MOLECULES THAT DECREASE THE ACTIVITY OF THE HUNTINGTIN GENE PROMOTER FOR THE POTENTIAL TREATMENT OF HUNTINGTON DISEASE(website)
- INHALED GM-CSF THERAPY OF AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS(website)
- INHIBITION OF AMA1-RON2 INTERACTION TO DISRUPT INVASION OF MALARIA PARASITE(website)
- IPGLYCERMIDES NOVEL POTENT AND SELECTIVE INHIBITORS OF PARASITIC PHOSPHOGLYCERATE MUTASE(website)
- KETOROLAC AND RELATED NSAIDS FOR TARGETING RHO-FAMILY GTPASES IN OVARIAN CANCER(website)
- LONG-ACTING PARATHYROID HORMONE ANALOG FOR THE TREATMENT OF HYPOPARATHYROIDISM(website)
- LUM-001 AS A TREATMENT FOR CREATINE TRANSPORTER DEFICIENCY(website)
- MODELING A RARE AUTONOMIC DISEASE(website)
- MODELING METABOLIC INTERACTIONS OF LIVER AND FAT WITH INTEGRATED MICROPHYSIOLOGICAL SYSTEMS(website)
- MODELLING RETT SYNDROME IN VASCULARIZED NEURAL ASSEMBLIES(website)
- NPC/MIPE TOXICITY SCREEN OF IDH1 INHIBITORS AGAINST CHOLANGIOCARCINOMA CELL LINES(website)
- PEROXISOME BIOGENESIS DISORDERS (PBDS)(website)
- PHENOTYPIC ASSAY DESIGN AND DEVELOPMENT FOR RARE AND NEGLECTED DISEASES(website)
- PHENOTYPIC TOXICITY SCREENING IN FAK/EWINGS SARCOMA(website)
- PORTABLE IRIDIUM-ELECTRODE INHALED NITRIC OXIDE GENERATOR FOR TREATMENT OF LUNG DISEASES(website)
- POTENTIAL THERAPEUTICS AGAINST ORTHOPOXVIRUSES(website)
- QHTS TO IDENTIFY ACTIVATORS OF E47(website)
- QHTS TO IDENTIFY INHIBITORS OF DYT1 INCLUSION FORMATION(website)
- QHTS TO IDENTIFY INHIBITORS OF NNMT1(website)
- QUANTUM MODEL REPURPOSING OF CETHROMYCIN FOR LIVER STAGE MALARIA(website)
- REDUCTION OF ATXN2 EXPRESSION AS A MEANS TO TREAT SPINOCEREBELLAR ATAXIA TYPE 2 (SCA2)(website)
- REPURPOSING AN EU THERAPEUTIC FOR HEMOGLOBINOPATHIES(website)
- REPURPOSING PYRONARIDINE AS A TREATMENT FOR THE EBOLA VIRUS(website)
- STUDIES OF TUMOR-PENETRATING MICROPARTICLES FOR PANCREATIC CANCER TRAUMA(website)
- THE DEVELOPMENT OF MULTIKINASE INHIBITORS FOR THE TREATMENT OF SELECTED CANCERS(website)
- TOXICOLOGY IN THE 21ST CENTURY PROGRAM (TOX21) – GENOMIC TOXICOLOGY(website)
- TREATMENT OF ACID CERAMIDASE DEFICIENCY(website)
- USE OF RAPAMYCIN FOR THE TREATMENT OF HYPERTROPHIC CARDIOMYOPATHY IN PATIENTS WITH LEOPARD SYNDROME(website)
2015
- 4TH INTERNATIONAL RASOPATHIES SYMPOSIUM(website)
- A TARGETED IMMUNOMODULATOR FOR THE TREATMENT OF ACUTE SICKLE CRISIS(website)
- ALS CLINICAL TRIALS GUIDELINES
- DYSTONIA COALITION(website)
- EVALUATION OF A CATHEPSIN S INHIBITOR AS A POTENTIAL DRUG FOR CHAGAS DISEASE(website)
- EVALUATION OF AZD9291 IN GLIOBLASTOMA PATIENTS WITH ACTIVATED EGFR(website)
- EXPANSION OF AN EFFICIENT DRUG REPURPOSING PLATFORM FOR RARE GENETIC DISEASES(website)
- FIBER DESIGN AND ASSESSMENT FOR DEVELOPMENT OF A NOVEL BIOMIMETIC MEDICAL DEVICE(website)
- RAPID POINT-OF-CARE DIAGNOSTIC FOR EARLY DETECTION OF DENGUE INFECTION(website)
- RAPID, NEAR-PATIENT TESTS TO MONITOR AND GUIDE THERAPY IN SICKLE CELL DISEASE PATIENTS
- RARE DISEASE RESEARCH TRAINING PROGRAM
- WEE1 AND HDAC INHIBITION IN RELAPSED/REFRACTORY AML(website)
2014
- ADMINISTRATIVE SUPPLEMENT: EXTRACELLULAR NON-CODING RNA BIOMARKERS OF HEPATOCELLULAR CANCER(website)
- BRITTLE BONE DISORDERS CONSORTIUM OF THE RARE DISEASE CLINICAL RESEARCH NETWORK
- DEVELOPMENT OF AN IPSC-DERIVED HUMAN VASCULAR SYSTEM FOR DRUG DISCOVERY AND DEVEL(website)
- FLUORESCENT RNA FOCI ASSAY FOR FXTAS DRUG DISCOVERY(website)
- FLUORESCENT RNA FOCI ASSAY FOR FXTAS DRUG DISCOVERY (website)
- HPV COMMUNICATION TO MICROENVIRONMENT VIA EXOSOMES(website)
- MOLECULAR LIBRARIES PROBE PRODUCTION CENTERS NETWORK(website)
- PHASE I, SBIR CONTRACT, N43TR-14-002?AN ASSAY TO MEASURE SURROGATE BIOMARKERS FOR DMD?
- PRECLINICAL INNOVATION(website)
- RARE DISEASES DATA MANAGEMENT AND COORDINATING CENTER(website)
2013
- A NEW CTSA PARTNERSHIP TO TRANSLATE AN ORAL CANCER BIOMARKER FROM LAB TO CLINIC (website)
- BIOCOMPUTATION ACROSS DISTRIBUTED PRIVATE DATASETS TO ENHANCE DRUG DISCOVERY(website)
- CLINICAL UTILITY OF SALIVARY EXRNA BIOMARKERS FOR GASTRIC CANCER DETECTION(website)
- COMB EXTERNAL BEAM RADIOTHERAPY WITH 153SM-EDTMP TO TREAT HIGH RISK OSTEOSARCOMA
- DEVELOPMENT OF A BIOCHEMICAL DIAGNOSIS FOR CREUTZFELDT-JAKOB DISEASE (website)
- ENZYME REPLACEMENT THERAPY FOR SANFILIPPO A LYSOSOMAL RARE DISEASE (website)
- EXOSOME BASED THERAPEUTICS IN HUNTINGTON’S DISEASE(website)
- EXRNA BIOMARKERS FOR HUMAN GLIOMA(website)
- EXTRACELLULAR NON-CODING RNA BIOMARKERS OF HEPATOCELLULAR CANCER(website)
- NOVEL EXTRA CELLULAR RNA-BASED COMBINATORIAL RNA INHIBITION THERAPY(website)
- PANCREATIC CANCER:ASSAY FOR EARLY DETECTION(website)
- PARTNERING TO TREAT AN ORPHAN DISEASE DUCHENNE MUSCULAR DYSTROPHY(website)
- PARTNERING TO TREAT AN ORPHAN DISEASE DUCHENNE MUSCULAR DYSTROPHY (website)
- PHARMACOLOGICAL SUPPRESSION OF ROD OPSIN AS THERAPY FOR RETINITIS PIGMENTOSA(website)
- PRECLINICAL DEVELOPMENT OF NOVEL MEK1/2 INHIBITORS TO TREAT INHERITED CARDIOMYOPA(website)
- TARGETED DELIVERY OF MICRORNA-LOADED MICROVESICLE FOR CANCER THERAPY(website)
- THERAPEUTIC STRATEGY FOR LAM (LYMPHANGIOLEIOMYOMATOSIS)(website)
2012
- A NEW CTSA PARTNERSHIP TO TRANSLATE AN ORAL CANCER BIOMARKER FROM LAB TO CLINIC(website)
- ANGELMAN SYNDROME (AS) (website)
- ASSESSING THE IMPACT OF OSTEOGENESIS IMPERFECTA ON NON-SKELETAL SYSTEMS (website)
- DRUG REPURPOSING AND REDISCOVERY FOR GASTROINTESTINAL STROMAL TUMORS(website)
- EARLY DETECTION OF PANCREATIC CANCER USING ICE COLD-PCR(website)
- MODELING OXIDATIVE STRESS AND DNA DAMAGE USING GI ORGANOTYPIC CULTURE SYSTEMS(website)
- ONLINE PATIENT SELF-ASSESSMENT SYSTEM FOR CARE AND RESEARCH OF JOINT AND SKIN DIS(website)
- PRADER-WILLI SYNDROME (PWS) (website)
- THERAPEUTICS FOR RARE AND NEGLECTED DISEASES – SCIENCE(website)
- WIRELESS AT-RISK INFANT MONITORING SYSTEM(website)
2011
- Drug Repurposing and Rediscovery for Gastrointestinal Stromal Tumors (website)