Grant List
Represents Grant table in the DB
GET /v1/grants?page%5Bnumber%5D=4&sort=-end_date
{ "links": { "first": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1&sort=-end_date", "last": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1424&sort=-end_date", "next": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=5&sort=-end_date", "prev": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=3&sort=-end_date" }, "data": [ { "type": "Grant", "id": "15961", "attributes": { "award_id": "1R35GM161641-01", "title": "Methods for quantifying selection and predicting evolutionary dynamics", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of General Medical Sciences (NIGMS)" ], "program_reference_codes": [], "program_officials": [ { "id": 44409, "first_name": "RONALD", "last_name": "ADKINS", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2026-03-01", "end_date": "2030-12-31", "award_amount": 417620, "principal_investigator": { "id": 44410, "first_name": "John P", "last_name": "Barton", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 3416, "ror": "", "name": "UNIVERSITY OF PITTSBURGH AT PITTSBURGH", "address": "", "city": "", "state": "PA", "zip": "", "country": "United States", "approved": true }, "abstract": "Natural selection is central to many challenges in biology and medicine, from the emergence of drug resistance in pathogens to cancer evolution. Understanding selection can also aid in protein engineering and help identify clinically relevant mutations in human disease genes. Temporal genetic data — sequences and phenotypes sampled over time — can be an especially powerful tool for understanding selection because it allows us to observe evolutionary dynamics directly. But while temporal data from sources like pathogen surveillance, ancient DNA, and experimental evolution have grown tremendously in recent years, statistical analyses of these data remain challenging. My lab will continue to pioneer the development of new computational methods to learn from temporal genetic data, revealing variants and phenotypes under selection and harnessing this information for predictive models of evolution. Over the past five years, we have developed several approaches to quantify selection from temporal data. Thanks to the use of mathematical methods from statistical physics, our methods are fast and accurate despite the inclusion of complex features such as linkage disequilibrium, epistasis, and time-varying selection. We demonstrated the power of these approaches through studies of HIV-1 immune escape and SARS-CoV-2 adaptation during the COVID-19 pandemic, where our analysis identified key mutations affecting viral transmission even before their importance was validated experimentally. Building on this foundation, we will pursue three synergistic research directions: First, we will develop new methods to jointly analyze selection on both individual mutations and phenotypic traits, fusing concepts from population genetics, quantitative genetics, and machine learning. Second, we will apply these methods to study rapid evolution in viral pathogens. Phenotypic models will help us to understand how immune pressure drives antigenic change in respiratory viruses and to compare evolutionary constraints on pathogens across host species. As an ambitious new direction, we will leverage these insights to develop predictive models of pathogen evolution, with influenza as a first target. Our research will systematically identify the features with the greatest power to predict evolution and characterize how and why predictive power may decline over time. Finally, we will extend our approaches to improve the interpretation of high-throughput mutagenesis experiments that measure the effects of thousands of mutations simultaneously. The proposed research will transform our understanding of how selection guides evolution across biological scales, from individual mutations to complex phenotypes, with applications ranging from predicting viral evolution to protein engineering. These advances could ultimately improve our ability to anticipate and control evolutionary processes across a wide range of biological contexts.", "keywords": [ "2019-nCoV", "Affect", "Biological", "Biology", "COVID-19 pandemic", "Complex", "Computing Methodologies", "DNA", "Data", "Data Sources", "Development", "Drug resistance", "Evolution", "Foundations", "Genes", "Genetic", "Genetic Epistasis", "Genotype", "HIV-1", "Immune", "Immunity", "Individual", "Influenza", "Learning", "Linkage Disequilibrium", "Machine Learning", "Malignant Neoplasms", "Maps", "Measures", "Medicine", "Methods", "Modeling", "Mutagenesis", "Mutation", "Natural Selections", "Phenotype", "Physics", "Play", "Population Genetics", "Process", "Protein Engineering", "Public Health", "Quantitative Genetics", "Research", "Resistance development", "Role", "Sampling", "Shapes", "Statistical Data Interpretation", "Statistical Methods", "Time", "Variant", "Viral", "clinically relevant", "experimental study", "genetic variant", "human disease", "improved", "insight", "mathematical methods", "pathogen", "pathogenic virus", "predictive modeling", "pressure", "respiratory virus", "tool", "trait", "viral transmission" ], "approved": true } }, { "type": "Grant", "id": "15971", "attributes": { "award_id": "1R35GM162359-01", "title": "The multifaceted pathways of astrovirus entry and egress", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of General Medical Sciences (NIGMS)" ], "program_reference_codes": [], "program_officials": [ { "id": 22244, "first_name": "MICHAEL", "last_name": "SAKALIAN", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2026-02-24", "end_date": "2030-12-31", "award_amount": 422677, "principal_investigator": { "id": 44425, "first_name": "Valerie", "last_name": "Cortez", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 3424, "ror": "", "name": "UNIVERSITY OF CALIFORNIA SANTA CRUZ", "address": "", "city": "", "state": "CA", "zip": "", "country": "United States", "approved": true }, "abstract": "Astroviruses are a major cause of pediatric diarrhea worldwide. Despite causing one of the most common early childhood infections, astroviruses are one of the least studied enteric RNA viruses. We previously discovered that the virus infects small intestinal goblet cells, specialized epithelial cells that secrete mucus. Few studies have investigated viral infection in goblet cells due to the lack of cell-specific models. Because the mechanisms by which viruses replicate inside of goblet cells are completely unknown, my lab is interested in addressing 1) how do astroviruses enter cells with highly dynamic apical membranes? and 2) what role does mucus secretion play in viral egress? We have established new in vitro models and tools to address these questions and have built a strong and collaborative investigative team with complementary expertise that will ensure the success of these projects. To evaluate receptor-mediated and fluid-phase endocytosis entry pathways into goblet cells, we will use a combination of CRISPR-Cas9 engineering, biochemical analysis, and high-resolution microscopy. We will use a similar suite of techniques as well as cryo-electron microscopy to define the egress pathway of astrovirus from goblet cells via mucus secretion. In addition to murine and human astroviruses, other respiratory and enteric viruses have also been shown to target goblet cells for infection. Thus, our work aims to initially provide foundational knowledge on the basic biology of astroviruses before shedding light on key host pathways in goblet cells that are co-opted by viruses from other families, including influenza and SARS-CoV2. Completion of these studies will provide the first major insights into the virus-host interactions at the apical membrane surface of intestinal goblet cells, which will pave the way for the future development of targeted drug treatments for the numerous viruses that target this unique cell population.", "keywords": [ "2019-nCoV", "Address", "Astrovirus", "Biochemical", "Biology", "CRISPR/Cas technology", "Cell model", "Cells", "Childhood", "Cryoelectron Microscopy", "Development", "Diarrhea", "Endocytosis", "Engineering", "Ensure", "Enteral", "Family", "Foundations", "Future", "Goblet Cells", "Human", "Infection", "Influenza", "Intestines", "Knowledge", "Light", "Liquid substance", "Mediating", "Microscopy", "Modeling", "Mucous body substance", "Mucus-Secreting Cell", "Mus", "Pathway interactions", "Phase", "Play", "Population", "Prevention strategy", "RNA Viruses", "Resolution", "Role", "Small Intestinal Goblet Cell", "Specialized Epithelial Cell", "Surface", "Techniques", "Viral", "Virus", "Virus Diseases", "Virus Replication", "Work", "apical membrane", "early childhood", "enteric virus infection", "in vitro Model", "insight", "interest", "novel", "receptor", "respiratory", "success", "targeted treatment", "tool", "treatment strategy", "virus host interaction" ], "approved": true } }, { "type": "Grant", "id": "15970", "attributes": { "award_id": "1R01DK145476-01", "title": "Mechanisms of non-HIV Collapsing Glomerulopathy in Hispanic Patients", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)" ], "program_reference_codes": [], "program_officials": [ { "id": 44424, "first_name": "KEVIN E", "last_name": "CHAN", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2026-02-15", "end_date": "2030-11-30", "award_amount": 783899, "principal_investigator": { "id": 22546, "first_name": "Sumant Singh", "last_name": "Chugh", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [ { "id": 804, "ror": "https://ror.org/01j7c0b24", "name": "Rush University Medical Center", "address": "", "city": "", "state": "IL", "zip": "", "country": "United States", "approved": true } ] }, "other_investigators": [], "awardee_organization": { "id": 3423, "ror": "", "name": "RUSH UNIVERSITY MEDICAL CENTER", "address": "", "city": "", "state": "IL", "zip": "", "country": "United States", "approved": true }, "abstract": "Non-HIV collapsing glomerulopathy (CG) is a severe form of glomerular disease seen in all parts of the world. Major etiological factors include circulating proteins that cause recurrent CG, infections like SARS-CoV-2 and Parvovirus B19, drugs like pamidronate, and background genomic changes potentially present in any of the preceding categories. Whereas variants of the APOL1 gene have been implicated in select populations with West African heritage, a genomic basis in other patient populations has not been elucidated. Using data from two decades of investigations into CG centered around Hispanic patients from Mexico City and more recent genomic data from Peruvian Hispanic patients, several novel mechanistic rat and mouse models of CG were developed. Critical components of the CG upstream pathways are the podocyte expressed transcriptional factor ZHX2 and glomerular integrins, including α3β1 in the podocyte, αvβ5 in the glomerular endothelium, and αvβ3 at both locations. Finally, recombinant mutated human Angiopoietin-like 4 protein 8520 is known to have an integrin stabilizing effect specific to Integrins β1 and β5, and could be potentially used to treat CG in the future. The overall premise of this application is that high podocyte ZHX2 expression and low glomerular endothelial Integrin β5 expression predispose to the development of CG. In Aim 1, changes in glomeruli with high podocyte ZHX2 expression will be investigated using transgenic rat models. Disease mechanism during the development of CG and just prior to the collapse of capillary loops will be elucidated. In Aim 2, changes in glomeruli with low endothelial Integrin β5 expression will be investigated using knockout mouse models. Disease mechanism during the development of CG and just prior to the collapse of capillary loops will be elucidated. In Aim 3, rat and mouse models of CG will be treated with protein 8520 to test for prevention or improvement in CG, and to potentially halt the disease process before the development of collapse.", "keywords": [ "10 year old", "2019-nCoV", "8 year old", "ANGPTL4 gene", "APOL1 gene", "Admixture", "Adriamycin PFS", "Affect", "African American", "Albuminuria", "Apoptosis", "Binding", "Binding Proteins", "Blood capillaries", "COVID-19 pandemic", "CRISPR/Cas technology", "Categories", "Cell Membrane Proteins", "Cell Nucleus", "Cell membrane", "Cells", "Cities", "Cytoplasmic Tail", "Data", "Development", "Disease", "Down-Regulation", "Endothelium", "Environment", "Ephrin-B1", "Etiology", "Event", "Feedback", "Focal and Segmental Glomerulosclerosis", "Foot Process", "Future", "Genetic", "Genomics", "Glomerular Capillary", "High Prevalence", "Hispanic", "Hispanic Populations", "Homeobox", "Homeodomain Proteins", "Human", "Human Parvovirus B19", "ITGB3 gene", "Incubated", "Infection", "Integrins", "Investigation", "Kidney Diseases", "Knockout Mice", "Location", "Membrane", "Mexico", "Modeling", "Mus", "Mutate", "Nuclear", "Pathogenesis", "Pathway interactions", "Patients", "Pattern", "Peruvian", "Pharmaceutical Preparations", "Phase", "Population", "Prevention", "Process", "Proliferating", "Proteins", "Proteinuria", "Publishing", "Rat Transgene", "Rattus", "Recombinants", "Recurrence", "Renal glomerular disease", "Rodent", "Role", "Series", "Serum", "Signal Transduction", "Site", "Sprague-Dawley Rats", "Testing", "Therapeutic Agents", "Therapeutic Effect", "Upregulation", "Variant", "Virus Diseases", "WT1 gene", "West African", "Zinc Fingers", "cytokine release syndrome", "diabetic", "exome", "genetic variant", "genomic data", "genotyped patients", "glomerular endothelium", "glomerular function", "glutamyl aminopeptidase", "improved", "in vivo", "induced pluripotent stem cell", "insertion/deletion mutation", "migration", "mouse model", "nephrotoxicity", "novel", "overexpression", "pamidronate", "pathogen", "patient population", "podocyte", "post SARS-CoV-2 infection", "prevent", "rat parvovirus", "slit diaphragm", "therapeutic evaluation", "transcription factor", "transmission process" ], "approved": true } }, { "type": "Grant", "id": "15963", "attributes": { "award_id": "1R01AG083894-01A1", "title": "Longitudinal MRI Measures of Cerebrovascular Injury and AD Atrophy in a Study of Latinos (SOL-INCA-MRI Long)", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute on Aging (NIA)" ], "program_reference_codes": [], "program_officials": [ { "id": 44413, "first_name": "MARYAM X", "last_name": "GHALEH", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2026-03-15", "end_date": "2030-11-30", "award_amount": 3517625, "principal_investigator": { "id": 44414, "first_name": "Charles", "last_name": "DeCarli", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 44415, "first_name": "Hector M", "last_name": "Gonzalez", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 3418, "ror": "", "name": "UNIVERSITY OF CALIFORNIA AT DAVIS", "address": "", "city": "", "state": "CA", "zip": "", "country": "United States", "approved": true }, "abstract": "Latinos constitute a heterogeneous population which accounted for slightly more than 50% of the United States (US) population growth for 2010 to 2020. Latinos are also becoming a larger proportion of older individuals in the US. Despite this, biomarker studies of normal aging and cognitive impairment remain limited, and the Hispanic Community Health Study/Study of Latinos (HCHS/SOL) constitutes the only known representative sample. Moreover, epidemiological data indicate that Latinos have a higher prevalence of vascular risk factors, lower cardiovascular health metrics, and a greater likelihood of having mild cognitive impairment (MCI) or dementia due non-Alzheimer’s disease processes that differ by heritage. Consequently, diagnosis and treatment of Latino persons with cognitive impairment may be more challenging, but more amenable to prevention through reduction of vascular risk factors than non-Hispanic White persons where vascular risk and disease is less prevalent. The first cycle of “MRI Measures of Cerebrovascular Injury and Alzheimer’s disease Atrophy in a Study of Latinos (RF1 AG054548; AKA SOL-INCA-MRI)” was designed to identify biological underpinnings of normal cognitive aging, MCI and Alzheimer’s disease and related dementias (ADRD) in a representative subgroup of the HCHS/SOL 62 + 9 years of age on average. Despite restrictions imposed by the COVID pandemic, our investigators successfully obtained brain MRI from 2668 individuals or >95% of the proposed study cohort. From these data we have published on 1) differences in brain structure from ages 35-85; 2) the impact of vascular risk and sleep on brain structure; 4) the association of cognition with subsequent MRI measures; and 4) genetic influences on select brain measures. These early results, while of scientific value, are cross- sectional and do not reflect ongoing degeneration or incident vascular injury, limiting inferential power that might extend scientific knowledge of brain aging and ADRD in this unique cohort. For this application, we propose to extend our work to include longitudinal MRI analysis, leveraging longitudinal biomarker and clinical data from 3 visits, spanning approximately 12 years of HCHS/SOL and its cognitive ancillary study (SOL-INCA- AD; R01 AG075758, Gonzalez, DeCarli Co-PIs) on a deeply characterized and diverse Hispanic/Latino cohort. Adding longitudinal image analysis in combination with longitudinal lifestyle, medical risk factors, plasma ATN biomarkers, genetics and cognitive assessment in this Latino cohort will address multiple ADRD research milestones and priorities while enabling stronger statistical inference of risk and resilience factors amongst representative, yet relatively young-old members of diverse Latino communities, creating the opportunity to identify modifiable risk factors, potentially reducing societal burden due to later-life ADRD in this rapidly growing portion of the older US population.", "keywords": [ "9 year old", "Age", "Aging", "Alzheimer's Disease", "Alzheimer's disease related dementia", "Amyloid beta-Protein", "Ancillary Study", "Atrophic", "Biological", "Biological Markers", "Blood Vessels", "Brain", "Brain Injuries", "COVID-19 pandemic", "Cerebrovascular Trauma", "Clinical Data", "Cognition", "Cognitive", "Cognitive aging", "Communities", "Data", "Dementia", "Diagnosis", "Disease", "Funding", "Genetic", "Genetic Risk", "Growth", "Hemorrhage", "High Prevalence", "Hispanic", "Hispanic Community Health Study/Study of Latinos", "Image Analysis", "Impaired cognition", "Individual", "Infarction", "Intercept", "K-Series Research Career Programs", "Knowledge", "Latino", "Latino Population", "Life", "Life Style", "Magnetic Resonance Imaging", "Measures", "Mediating", "Medical", "Nerve Degeneration", "Not Hispanic or Latino", "Older Population", "Participant", "Pathologic", "Persons", "Plasma", "Population", "Population Growth", "Population Heterogeneity", "Prevention", "Process", "Publishing", "Research", "Research Personnel", "Risk", "Risk Factors", "Sampling", "Sleep", "Structure", "Study of Latinos", "Subgroup", "Time", "United States", "United States National Institutes of Health", "Vascular Diseases", "Visit", "Water", "White Matter Hyperintensity", "Work", "aging brain", "brain magnetic resonance imaging", "cardiovascular health", "cerebral microbleeds", "cognitive performance", "cognitive testing", "cohort", "cohort research", "design", "disparity reduction", "endophenotype", "epidemiologic data", "gray matter", "magnetic resonance imaging biomarker", "member", "mild cognitive impairment", "modifiable risk", "morphometry", "normal aging", "resilience factor", "serial imaging", "social culture", "sociocultural determinant", "tau-1", "vascular injury", "vascular risk factor", "white matter" ], "approved": true } }, { "type": "Grant", "id": "15826", "attributes": { "award_id": "3R01AI190528-01S3", "title": "FEND-TB", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Allergy and Infectious Diseases (NIAID)" ], "program_reference_codes": [], "program_officials": [ { "id": 44239, "first_name": "NICOLE JOY", "last_name": "ESPY", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-09-10", "end_date": "2030-08-31", "award_amount": 298587, "principal_investigator": { "id": 8100, "first_name": "David", "last_name": "Alland", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [ { "id": 925, "ror": "", "name": "RBHS-NEW JERSEY MEDICAL SCHOOL", "address": "", "city": "", "state": "NJ", "zip": "", "country": "United States", "approved": true } ] }, "other_investigators": [ { "id": 27206, "first_name": "Jerrold J.", "last_name": "Ellner", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, { "id": 44240, "first_name": "Susan E", "last_name": "Dorman", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 1856, "ror": "", "name": "RUTGERS BIOMEDICAL AND HEALTH SCIENCES", "address": "", "city": "", "state": "NJ", "zip": "", "country": "United States", "approved": true }, "abstract": "Among the estimated 10 million people with tuberculosis (TB) in 2022, over 3 million were not diagnosed, underscoring the need for new tools and diagnostic strategies to improve TB case detection, including in difficult to-diagnose populations such as children and people with extrapulmonary or early/subclinical TB. An equally urgent need is for drug susceptibility tests (DST) that can rapidly identify TB drug resistance/susceptibility, including to new drugs, to cure each person with TB and to protect the efficacy of newer drugs for those who may benefit from them in the future. Partially fueled by innovative platforms developed to address the COVID-19 epidemic, the last decade has seen a surge of new TB diagnostic platforms and assays. These new tests, sometimes developed by small companies with limited resources and little or no experience working with Mycobacterium tuberculosis (MTB) and clinical TB, require rigorous and unbiased evaluations. Furthermore, many diagnostic developers do not sufficiently appreciate the challenges presented by sample processing, the (often) need for high test sensitivity, or the acceptable tradeoffs between sensitivity/specific versus assay costs, ease of use and relevant target populations or TB disease states. Thus, the unmet needs of TB diagnostic developers are specific to the type of manufacturer, diagnostic technology, assay format, and intended use. Our proposal brings together a consortium of experienced investigators and a global network of clinical research sites to support TB diagnostic evaluations through a fast-paced and flexible pipeline that includes tech scouting, development mentoring, and clinical testing of early-stage TB diagnostics. With the inclusion of an Analytic Laboratory Core that can assess and help optimize new technologies prior to and after clinical studies our program offers a unique opportunity to accelerate promising TB diagnostics through their optimal development pipeline. Consortium members have almost three decades of leadership experience in diagnostic tech scouting, research, development, clinical trialing, and implementation activities. Fielding a balance of established platforms, fast followers, and cutting- edge technologies, we will perform the following specific aims: 1) identify promising early-stage diagnostics for evaluation and develop for each a stepwise evaluation plan; 2) evaluate the diagnostic accuracy of novel rapid point of care/near care TB diagnostics and determine the impact of relevant patient characteristics on test accuracy; and 3) provide feedback to diagnostic developers, policymakers, and other stakeholders on performance and usability of novel diagnostic tests and potential strategies for use in TB endemic settings.", "keywords": [ "Acceleration", "Address", "Adult", "Affect", "Biological Assay", "Biological Markers", "COVID-19 pandemic", "Caring", "Cause of Death", "Characteristics", "Child", "Childhood", "Clinical", "Clinical Research", "Clinical Trials", "Clinical assessments", "Communicable Diseases", "Communities", "Country", "Detection", "Development", "Diagnosis", "Diagnostic", "Diagnostic tests", "Diagnostics Research", "Disease", "Drug resistance", "Drug resistance in tuberculosis", "Enrollment", "Ensure", "Equilibrium", "Evaluation", "Feedback", "Funding", "Future", "HIV/TB", "Health", "India", "International", "Laboratories", "Leadership", "Manufacturer", "Mentors", "Mentorship", "Moldova", "Mycobacterium tuberculosis", "Patient Care", "Patient Recruitments", "Patient-Focused Outcomes", "Patients", "Performance", "Persons", "Peru", "Pharmaceutical Preparations", "Policy Maker", "Population", "Predisposition", "Process", "Reporting", "Research", "Research Personnel", "Resources", "Sampling", "South Africa", "Specificity", "Specimen", "Sputum", "Symptoms", "Target Populations", "Technology", "Testing", "Time", "Tuberculosis", "Tuberculosis diagnosis", "Uganda", "United States National Institutes of Health", "Vietnam", "Work", "biobank", "clinical diagnostics", "clinical research site", "comorbidity", "cost", "design", "diagnostic accuracy", "diagnostic assay", "diagnostic platform", "diagnostic strategy", "diagnostic technologies", "drug testing", "experience", "flexibility", "improved", "innovation", "member", "new technology", "novel", "novel diagnostics", "novel therapeutics", "performance site", "point of care", "precision medicine", "programs", "prospective", "prototype", "rapid test", "research clinical testing", "response", "tool", "treatment response", "tuberculosis diagnostics", "tuberculosis treatment", "usability" ], "approved": true } }, { "type": "Grant", "id": "12061", "attributes": { "award_id": "1U24HL166784-01A1", "title": "2/2: PREcision VENTilation to attenuate Ventilation-Induced Lung Injury (PREVENT VILI)", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Heart Lung and Blood Institute (NHLBI)" ], "program_reference_codes": [], "program_officials": [ { "id": 22589, "first_name": "CHRISTIAN RENE", "last_name": "Gomez", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2023-09-01", "end_date": "2030-08-31", "award_amount": 1175434, "principal_investigator": { "id": 27913, "first_name": "Elias", "last_name": "Baedorf Kassis", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 27914, "first_name": "TIMOTHY T", "last_name": "HOULE", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 771, "ror": "https://ror.org/04drvxt59", "name": "Beth Israel Deaconess Medical Center", "address": "", "city": "", "state": "MA", "zip": "", "country": "United States", "approved": true }, "abstract": "/ ABSTRACT Acute respiratory distress syndrome (ARDS) occurs in up to one-quarter of all critically ill adults receiving mechanical ventilation and is associated with high risk of morbidity and mortality, which is compounded by ventilation-induced lung injury (VILI). Current ventilation strategies and standard care attempt to lessen the effects of VILI, but have not been uniformly successful. The need for improved ventilation strategies have become more compelling and urgent in the context of the COVID-19 pandemic. Precise ventilator titration of tidal volumes to maintain driving pressure ≤ 12 cm H2O may improve overdistension injury and application of positive end expiratory pressure (PEEP) with titration to transpulmonary pressure of 0±2cmH2O may prevent injury from lung collapse. This U24 grant application will establish a Data Coordinating Center (DCC) that supports the PREcision VENTilation to attenuate VILI Clinical Coordinating Center (CCC) in conducting a multicenter, prospective phase III randomized trial to test the hypothesis that this precision ventilation strategy will improve 60-day mortality compared to guided usual care in patients with moderate or severe ARDS. The DCC will provide the infrastructure, support, oversight and quality control necessary to conduct this trial guided by the following aims: Aim 1 - To collaborate in the protocol development. The DCC will assist the CCC in defining the overarching plan of the trial, and ensure the inclusion of essential sections within the protocol document. Aim 2 - To design and execute an appropriate, pre-determined, innovative statistical plan including analysis of study data, assessment of safety, investigation of mechanism of benefit, and facilitation of manuscript preparation. Aim 3 – To facilitate a patient centered study, with community engagement, by providing video-assisted consent materials, real-time reporting of accrual rates, with emphasis on ensuring a diverse participant population and ensuring patient safety by providing clear instructions on identification and timing of adverse event reporting and creation of the Data and Safety Monitoring Board. Aim 4 - To support an advanced physiological core laboratory to integrate physiological expertise into trial conduct and data management and to ensure rigor and reproducibility of respiratory waveform interpretation used for protocol-directed ventilator management in the intervention arm. The DCC will review all physiological waveforms submitted by sites and provide feedback regarding quality and interpretation. Aim 5 - To provide data administration and monitor trial activities. The DCC will build the eCRFs on a state-of-the-art electronic data capture system. During enrollment, the DCC will ensure high-quality data collection and measure and improve protocol compliance. To foster site communication and identity, the DCC will develop a trial website and quarterly newsletter, and assist the CCC in organizing meetings and conference calls. Aim 6 - To ensure completeness of the study and post-trial activities. This includes meeting milestones and end-of-trial support for participating sites and providing the funding agency with a finalized, deidentified, and locked data set for public use.", "keywords": [ "Acute Respiratory Distress Syndrome", "Address", "Admission activity", "Adult", "Adverse event", "Applications Grants", "Attenuated", "Automobile Driving", "Biological Markers", "Body Weight", "Breathing", "COVID-19 mortality", "COVID-19 pandemic", "Case Report Form", "Cause of Death", "Clinical", "Clinical Trials Data Monitoring Committees", "Collaborations", "Communication", "Consent", "Critical Illness", "Data", "Data Collection", "Data Coordinating Center", "Data Set", "Electronics", "Enrollment", "Ensure", "Feedback", "Fostering", "Funding Agency", "Goals", "Grant", "Healthcare Systems", "Infrastructure", "Injury", "Instruction", "Intervention", "Investigation", "Laboratories", "Lung", "Maintenance", "Manuscripts", "Measurement", "Measures", "Mechanical ventilation", "Methodology", "Monitor", "Morbidity - disease rate", "Newsletter", "Outcome", "Participant", "Patients", "Phase", "Physiological", "Population", "Positive-Pressure Respiration", "Preparation", "Prevention strategy", "Protocol Compliance", "Protocols documentation", "Quality Control", "Randomized Controlled Trials", "Recording of previous events", "Recovery", "Reporting", "Reproducibility", "Research", "Research Personnel", "Respiratory Mechanics", "Risk", "Shock", "Site", "Stress", "Techniques", "Teleconferences", "Testing", "Tidal Volume", "Time", "Titrations", "Ventilator", "Ventilator-induced lung injury", "arm", "atelectrauma", "community engagement", "cost", "data management", "design", "efficacy testing", "electronic data capture system", "evidence base", "expiration", "hemodynamics", "high risk", "high standard", "improved", "innovation", "lung injury", "lung pressure", "meetings", "mortality", "novel", "patient oriented", "patient safety", "personalized approach", "personalized strategies", "pre-pandemic", "pressure", "prevent", "primary outcome", "prospective", "protocol development", "randomized trial", "respiratory", "safety assessment", "standard care", "standard of care", "statistical center", "support tools", "symposium", "treatment arm", "treatment as usual", "trial planning", "usual care arm", "ventilation", "web site" ], "approved": true } }, { "type": "Grant", "id": "15883", "attributes": { "award_id": "1K24AI187743-01", "title": "Determinants of Vaccine Responses in Low-Income Countries (LICs)", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Allergy and Infectious Diseases (NIAID)" ], "program_reference_codes": [], "program_officials": [ { "id": 32597, "first_name": "BROOKE ALLISON", "last_name": "BOZICK", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-09-01", "end_date": "2030-08-31", "award_amount": 176738, "principal_investigator": { "id": 7688, "first_name": "Clarissa", "last_name": "Valim", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 860, "ror": "", "name": "BOSTON UNIVERSITY MEDICAL CAMPUS", "address": "", "city": "", "state": "MA", "zip": "", "country": "United States", "approved": true }, "abstract": "By mentoring junior investigators from the United States and low-come countries (LICs) in patient- oriented research (POR) in global health, particularly in vaccine-preventable diseases, I hope to build local capacity, improve outcomes and reduce health disparities. Working within the resource-limited health systems in LICs is challenging, and the influences of co-infections (particularly parasitic infections), anemia, undernutrition, and various microorganisms on vaccine efficacy may be underappreciated. For these reasons, vaccines and vaccine campaigns developed for and assessed in high-income countries (HICs) cannot be assumed to be feasibly implemented or to perform similarly in LICs. For instance, SARS-CoV-2 vaccination with mRNA-based vaccines in sub-Saharan Africa has been limited by the cold-chain requirement of these vaccines. Cultural and socio-economic factors have led Malawians to generally choose receiving the single dose Ad26.COV2-S rather than the ChAdOx1 vaccine. Growing evidence suggests that performance of vaccines in LICs is lower than HICs and the protective immunity elicited by some vaccines is shorter than originally anticipated. In an interconnected world where diseases do not have borders, optimizing interventions to control transmittable diseases in LICs impacts the health of populations worldwide. The overall goal is to train junior investigators across disciplines to optimize vaccinations in Malawi, a sub-Saharan African country in which I have established a thriving research infrastructure. The first two aims of this application, supported by my R01AI164686-funded project, will assess i) the longevity of antibodies (magnitude and function) and memory cells induced by the Ad26.COV2-S SARS-CoV-2 vaccine; ii) the contribution of pre-existing changes in innate immunity to these responses; iii) the association of malaria, micronutrient deficiency, and microorganisms colonizing the nasopharynx with these responses. In the third aim, funded through this proposal, we will address the longevity of five childhood vaccines, focusing particularly on optimizing the timing of re-vaccination. The importance of this question became obvious in 2023 when three poliovirus cases were identified in Malawi. The Ministry of Health, with no relevant data available, decided to revaccinate all children with an arbitrary cutoff of 15 years of age. I have mentored several investigators in POR, particularly from Malawi, and have been conducting research in the immunoepidemiology of vaccines in LICs (including Malawi) since 2015. With this award, I would be able to expand my panel of trainees, devote more time to each of them, acquire training to further my mentorship skills as well as my own professional skillset, and expand the focus of the research to include other childhood vaccines. With the research infrastructure built in Malawi, I am now well-equipped to successfully train clinician scientists and other junior investigators in POR in infectious diseases, particularly in vaccines in LICS.", "keywords": [ "10 year old", "15 year old", "2019-nCoV", "Adjuvant", "Affect", "Africa South of the Sahara", "African", "American", "Anemia", "Antibiotic Resistance", "Antibodies", "Antibody Response", "Award", "B-Lymphocytes", "Bacteria", "Binding", "COVID-19 morbidity", "COVID-19 vaccination", "COVID-19 vaccine", "Cells", "Child", "Childhood", "Clinical Investigator", "Cold Chains", "Communicable Diseases", "Contracts", "Country", "Data", "Deceleration", "Dedications", "Development", "Discipline", "Disease", "Dose", "Exposure to", "Fever", "Fostering", "Frequencies", "Funding", "Goals", "Grant", "Haemophilus influenzae type b", "Health", "Health system", "Human", "Human poliovirus", "Immune response", "Immunity", "Immunologic Epidemiology", "Income", "Individual", "Infection", "Innate Immune Response", "Intervention", "Knowledge", "Lead", "Longevity", "Malaria", "Malaria Vaccines", "Malawi", "Malnutrition", "Measles", "Measles Vaccine", "Memory", "Mentors", "Mentorship", "Micronutrients", "Mid-Career Clinical Scientist Award (K24)", "Morbidity - disease rate", "Nasopharynx", "Natural Immunity", "Oropharyngeal", "Parasitic infection", "Performance", "Pertussis", "Pertussis Vaccine", "Pilot Projects", "Policies", "Population", "RNA vaccine", "Reduce health disparities", "Research", "Research Infrastructure", "Research Personnel", "Resources", "Risk", "Risk Factors", "Rotavirus", "Rotavirus Vaccines", "SARS-CoV-2 infection", "SARS-CoV-2 transmission", "Sahara", "School-Age Population", "Scientist", "Severities", "Socioeconomic Factors", "Supplementation", "Tetanus", "Tetanus Vaccine", "Time", "Training", "United States", "Vaccination", "Vaccinee", "Vaccines", "Virus", "Work", "Zinc deficiency", "adaptive immune response", "adaptive immunity", "antibody-dependent cell cytotoxicity", "biological systems", "booster vaccine", "career", "co-infection", "global health", "improved outcome", "interest", "international center", "iron deficiency", "low income country", "malaria infection", "microbiome", "micronutrient deficiency", "microorganism", "mortality", "novel vaccines", "pathogen", "patient oriented research", "population health", "prevent", "programs", "response", "skills", "vaccine efficacy", "vaccine response" ], "approved": true } }, { "type": "Grant", "id": "15889", "attributes": { "award_id": "1R01AI194296-01", "title": "Spatial and Temporal Adjuvant Delivery for Skin mRNA Vaccination", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Allergy and Infectious Diseases (NIAID)" ], "program_reference_codes": [], "program_officials": [ { "id": 32831, "first_name": "JENNIFER L", "last_name": "GORDON", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2025-09-03", "end_date": "2030-08-31", "award_amount": 676666, "principal_investigator": { "id": 24143, "first_name": "Mei X", "last_name": "Wu", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [ { "id": 736, "ror": "https://ror.org/002pd6e78", "name": "Massachusetts General Hospital", "address": "", "city": "", "state": "MA", "zip": "", "country": "United States", "approved": true } ] }, "other_investigators": [], "awardee_organization": { "id": 2623, "ror": "", "name": "MASSACHUSETTS GENERAL HOSPITAL", "address": "", "city": "", "state": "MA", "zip": "", "country": "United States", "approved": true }, "abstract": "The success of COVID-19 mRNA vaccines has showcased the effectiveness of this technology in rapid design, manufacturing, and adaptation to emerging viral threats. However, several imitations remain, including their instability requiring cold-chain storage that limits their global distribution, the necessity for two doses for optimal efficacy, limited immunity to closely matched viral strains, and quick-waning immunity following the initial two doses. These limitations can be potentially addressed by potent adjuvants. However, traditional adjuvants, co-delivered with vaccines to enhance antigen-presenting cell (APC) recruitment and functionality, are unsuitable for mRNA vaccines, because the co-delivered adjuvant provokes innate immune responses that interfere with mRNA translation, reducing its immunogenicity. Our recent investigation demonstrated that mRNA vaccines, particularly when delivered intradermally, could be substantially augmented by adjuvant delivery at different times and locations relative to mRNA vaccination, effectively circumventing the interference with mRNA transcription and translation. The current proposal aims to determine whether intranasal (IN) or intradermal (ID) delivery of a newly developed adjuvant can expand the breadth and longevity of trivalent influenza (flu) mRNA vaccine administered via a microneedle array patch (MNP). Specifically, we will construct a trivalent self-amplifying mRNA (saRNA) flu vaccine, encapsulated within liposomes free of cationic or ionizable lipids using our proprietary technology, and then loaded into our caved MNP after lyophilization. The antigen expression, immunogenicity, and efficacy of this saRNA flu vaccine will be optimized and evaluated in mice. Aim 2 will define the optimal time window of IN or ID adjuvant delivery following MNP-saRNA- flu immunization. Antigens-specific T-cell responses and broad neutralization antibodies across homosubtypic and heterosubtypic flu strains will be assessed and compared with or without the adjuvant and between IN and ID adjuvant delivery. The longevity of the specific immune responses will be also monitored over several months. Aim 3 will validate the efficacy of MNP immunization, alongside delayed adjuvant delivery, against various flu viruses in young and aging mice. If successful, the innovative strategy can be extended to other respiratory viral vaccines, such as those for COVID-19, respiratory syncytial viruses, etc., broadening the breadth and efficacy of various RNA-based vaccines delivered parenterally.", "keywords": [ "Acute", "Address", "Adjuvant", "Adjuvanticity", "Adverse event", "Aging", "Agonist", "Antigen-Presenting Cells", "Antigens", "B-Lymphocytes", "Biomimetics", "COVID-19", "Cellular Immunity", "Cold Chains", "Coupled", "Custom", "Death Rate", "Distant", "Dose", "Effectiveness", "Encapsulated", "Epithelial Cells", "Flu virus", "Freeze Drying", "Genetic Transcription", "Grant Review", "Hemagglutinin", "Homing", "Immune", "Immune response", "Immunity", "Immunization", "Immunize", "Inferior", "Inflammation", "Influenza", "Influenza A Virus H3N2 Subtype", "Influenza vaccination", "Innate Immune Response", "Intramuscular", "Investigation", "Lipids", "Liposomes", "Location", "Longevity", "Lung", "Lymph", "Mediating", "Messenger RNA", "Monitor", "Mucous Membrane", "Mus", "Mutation", "Natural Immunity", "Needles", "Nuclear", "Proteins", "Pulmonary Surfactants", "RNA vaccination", "RNA vaccine", "Respiratory syncytial virus", "Risk", "Safety", "Science", "Self Efficacy", "Skin", "Stimulator of Interferon Genes", "T cell response", "T-Lymphocyte", "Technology", "Temperature", "Time", "Tissues", "Toxic effect", "Translations", "Vaccination", "Vaccines", "Viral", "Viral Antigens", "Viral Vaccines", "Virus", "alveolar epithelium", "antigen-specific T cells", "clinically significant", "cross immunity", "cross reactivity", "design", "efficacy validation", "flu", "immunogenicity", "in vivo", "influenza virus strain", "influenza virus vaccine", "influenzavirus", "innovation", "lipid nanoparticle", "mRNA Translation", "mRNA delivery", "manufacture", "mortality", "nanoparticle", "neutralizing antibody", "old mice", "pandemic disease", "rapid growth", "recruit", "respiratory", "respiratory pathogen", "spatiotemporal", "success", "translational potential", "vaccine delivery", "vaccine efficacy" ], "approved": true } }, { "type": "Grant", "id": "15437", "attributes": { "award_id": "5U24HL166784-02", "title": "2/2: PREcision VENTilation to attenuate Ventilation-Induced Lung Injury (PREVENT VILI)", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Heart Lung and Blood Institute (NHLBI)" ], "program_reference_codes": [], "program_officials": [ { "id": 22589, "first_name": "CHRISTIAN RENE", "last_name": "Gomez", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2023-09-01", "end_date": "2030-08-31", "award_amount": 661149, "principal_investigator": { "id": 27913, "first_name": "Elias", "last_name": "Baedorf Kassis", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 27914, "first_name": "TIMOTHY T", "last_name": "HOULE", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 771, "ror": "https://ror.org/04drvxt59", "name": "Beth Israel Deaconess Medical Center", "address": "", "city": "", "state": "MA", "zip": "", "country": "United States", "approved": true }, "abstract": "/ ABSTRACT Acute respiratory distress syndrome (ARDS) occurs in up to one-quarter of all critically ill adults receiving mechanical ventilation and is associated with high risk of morbidity and mortality, which is compounded by ventilation-induced lung injury (VILI). Current ventilation strategies and standard care attempt to lessen the effects of VILI, but have not been uniformly successful. The need for improved ventilation strategies have become more compelling and urgent in the context of the COVID-19 pandemic. Precise ventilator titration of tidal volumes to maintain driving pressure ≤ 12 cm H2O may improve overdistension injury and application of positive end expiratory pressure (PEEP) with titration to transpulmonary pressure of 0±2cmH2O may prevent injury from lung collapse. This U24 grant application will establish a Data Coordinating Center (DCC) that supports the PREcision VENTilation to attenuate VILI Clinical Coordinating Center (CCC) in conducting a multicenter, prospective phase III randomized trial to test the hypothesis that this precision ventilation strategy will improve 60-day mortality compared to guided usual care in patients with moderate or severe ARDS. The DCC will provide the infrastructure, support, oversight and quality control necessary to conduct this trial guided by the following aims: Aim 1 - To collaborate in the protocol development. The DCC will assist the CCC in defining the overarching plan of the trial, and ensure the inclusion of essential sections within the protocol document. Aim 2 - To design and execute an appropriate, pre-determined, innovative statistical plan including analysis of study data, assessment of safety, investigation of mechanism of benefit, and facilitation of manuscript preparation. Aim 3 – To facilitate a patient centered study, with community engagement, by providing video-assisted consent materials, real-time reporting of accrual rates, with emphasis on ensuring a diverse participant population and ensuring patient safety by providing clear instructions on identification and timing of adverse event reporting and creation of the Data and Safety Monitoring Board. Aim 4 - To support an advanced physiological core laboratory to integrate physiological expertise into trial conduct and data management and to ensure rigor and reproducibility of respiratory waveform interpretation used for protocol-directed ventilator management in the intervention arm. The DCC will review all physiological waveforms submitted by sites and provide feedback regarding quality and interpretation. Aim 5 - To provide data administration and monitor trial activities. The DCC will build the eCRFs on a state-of-the-art electronic data capture system. During enrollment, the DCC will ensure high-quality data collection and measure and improve protocol compliance. To foster site communication and identity, the DCC will develop a trial website and quarterly newsletter, and assist the CCC in organizing meetings and conference calls. Aim 6 - To ensure completeness of the study and post-trial activities. This includes meeting milestones and end-of-trial support for participating sites and providing the funding agency with a finalized, deidentified, and locked data set for public use.", "keywords": [], "approved": true } }, { "type": "Grant", "id": "15818", "attributes": { "award_id": "1U54HD121579-01", "title": "Project 3- Access Study", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)" ], "program_reference_codes": [], "program_officials": [], "start_date": "2025-09-15", "end_date": "2030-08-31", "award_amount": 204764, "principal_investigator": { "id": 44226, "first_name": "Kimberly Ann", "last_name": "Chapman", "orcid": "", "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 2610, "ror": "", "name": "BAYLOR COLLEGE OF MEDICINE", "address": "", "city": "", "state": "TX", "zip": "", "country": "United States", "approved": true }, "abstract": "7. ABSTRACT – PROJECT 3 (TELEHEALTH VS VIRTUAL VISITS IN ORGANIC ACIDEMIAS) In a recent survey from the National Organization for Rare Disorders (NORD), nearly 40% of patients/caregivers with rare disorders reported traveling more than 60 miles for medical care. The same survey also found that 70% of respondents would like the option of telehealth for medical appointments and that during the SARS-CoV-2 pandemic, 88% of those offered a video appointment accepted. Of those accepting a video visit, 92% said it was a positive experience. In addition to the access to care issues, the NORD survey also found that 62% of patients with rare diseases were not able to attend work due to their appointments, and that 26% of children missed school regularly for appointments. Consequently, NORD is strongly advocating robust access to telehealth services for patients with rare diseases. Access to care for rare organic acidemias is an even greater challenge. In a recent survey by the Organic Acidemia Association and Propionic Acidemia Foundation, access to care was a top concern for individuals and families. According to the American Board of Medical Genetics and Genomics website, ten US states have no provider certified in either clinical or medical biochemical genetics demonstrating a significant gap in access to local specialists with expertise in organic acidemias. Although telemedicine has been shown to be effective for genetic counseling and genetic patient evaluations, care for individuals with organic acidemias involves more than counseling and diagnosis. Treatment for organic acidemias is complex requiring a combination of low protein diets, medications/supplements, strict adherence to treatment regimens, and the implementation of sick-day protocols in collaboration with the healthcare team. Frequent monitoring of growth, nutrition, and laboratory values and ongoing education are critical to successfully managing organic acidemias. We hypothesize that telehealth is one strategy for increasing access to high-quality care for organic acidemias. To test this hypothesis, we will perform the first large, multi-center study evaluating the efficacy of telehealth for organic acidemias with the following aims: 1) Assess whether virtual clinic visits are equivalent to in-person visits at achieving treatment goals, 2) Compare attendance at clinic visits and knowledge of treatment regimens for those receiving care at virtual and in-person visits, and 3) Assess patient and parent satisfaction and patient-care team relationships with virtual clinic visits compared to in-person visits. Overall, this multi-center, longitudinal study of virtual vs. in-person medical visits will demonstrate whether virtual visits are effective at providing high quality care for individuals with these disorders. As virtual visits may be a strategy for increasing access to clinical trials and meeting enrollment goals in trials for organic acidemias, the results of this study are important for both clinical trial readiness and improving patient access to quality care.", "keywords": [ "Acute", "Adherence", "Advocate", "American", "Appointment", "Area", "Biochemical", "Biochemical Genetics", "COVID-19 pandemic", "Caregivers", "Caring", "Certification", "Child", "Client satisfaction", "Clinic Visits", "Clinical", "Clinical Trials", "Collaborations", "Collection", "Complex", "Counseling", "Diagnosis", "Diet", "Disease", "Education", "Emergency department visit", "Enrollment", "Evaluation", "Event", "Family", "Foundations", "Genetic", "Genetic Counseling", "Genomics", "Goals", "Growth", "Health Services", "Health Services Accessibility", "Hospitalization", "Illness Days", "Improve Access", "Inborn Errors of Metabolism", "Individual", "Knowledge", "Laboratories", "Logistics", "Longitudinal Studies", "Measures", "Medical", "Medical Care Team", "Medical Genetics", "Metabolic", "Monitor", "Multicenter Studies", "Nutrition", "Outcome", "Outcome Assessment", "Outpatients", "Parents", "Participant", "Patient Care Team", "Patients", "Persons", "Pharmaceutical Preparations", "Protein-Restricted Diet", "Protocols documentation", "Provider", "Quality of Care", "Randomized", "Rare Diseases", "Regimen", "Reporting", "Research", "Respondent", "Schools", "Specialist", "Structure", "Surveys", "Telemedicine", "Testing", "Travel", "Treatment Protocols", "US State", "Visit", "Work", "acute care", "care providers", "clinical trial readiness", "dietary", "efficacy evaluation", "experience", "gaps in access", "genetics clinic", "improved", "improved outcome", "ketotic hyperglycinemia", "medical appointment", "meetings", "metropolitan", "patient advocacy group", "satisfaction", "telehealth", "therapeutically effective", "video visit", "virtual", "virtual visit", "web site" ], "approved": true } } ], "meta": { "pagination": { "page": 4, "pages": 1424, "count": 14236 } } }