grant
Project 2: Improved Presymptomatic Diagnosis in Aicardi Goutieres Syndrome
Organization CHILDREN'S HOSP OF PHILADELPHIALocation PHILADELPHIA, UNITED STATESPosted 30 Sept 2019Deadline 31 Aug 2030
NIHUS FederalResearch GrantFY2025(IFN) α(IFN)-α(IFN)α0-11 years old0-4 weeks oldAddison disease-cerebral sclerosis syndromeAddison disease-spastic paraplegia syndromeAddison-Schilder syndromeAdrenoleukodystrophyAdvocacyAffectAicardi Goutieres syndromeAlferonAntigensAssayBindingBioassayBiological AssayBiological MarkersBirthBlindedBloodBlood Reticuloendothelial SystemBlood ScreeningBronze Schilder diseaseCREE ENCEPHALITISCell Communication and SignalingCell SignalingChildChild YouthChildren (0-21)Clinical ResearchClinical StudyClinical Trials NetworkCognitive DiscriminationCollaborationsCoupledDNA metabolismDataDetectionDevelopmentDiagnosisDiagnosticDiagnostic SensitivityDiagnostic SpecificityDiscriminationDiseaseDisorderDissociationELISAEarly DiagnosisEarly InterventionEncephalopathy with intracranial calcification and chronic lymphocytosis of cerebrospinal fluidEncephalopathy, familial infantile, with calcification of basal ganglia and chronic cerebrospinal fluid lymphocytosisEnzyme-Linked Immunosorbent AssayFanconi-Prader syndromeFrequenciesFutureG1P3Gene CombinationsGene ExpressionGene Expression MonitoringGene Expression Pattern AnalysisGene Expression ProfilingGenesGeneticGenotypeGoalsHereditary DiseaseIFI6IFI6 geneIFNIFN AlphaIFN activationIFN αIFN-αIFNaIFNαImmunoassayInborn Genetic DiseasesIncidenceInfectionInflammatoryInherited disorderInterferon ActivationInterferon Alfa-n3Interferon-αInterferonsIntracellular Communication and SignalingJAK kinaseJanus kinaseLanthanidesLanthanoid Series ElementsLanthanoidsLeukocyte InterferonLifeLinkLive BirthLupus Erythematosus DisseminatusLymphoblast InterferonLymphoblastoid InterferonMeasurableMeasurementMessenger RNAMolecular InteractionMorbidityMorbidity - disease rateMusculoskeletal Pain DisorderNatural HistoryNeonatalNeonatal ScreeningNervous System InjuriesNervous System TraumaNervous System damageNeurologicNeurologicalNeurological DamageNeurological InjuryNeurological traumaNewborn InfantNewborn Infant ScreeningNewbornsNon-Polyadenylated RNAOutcomeParturitionPathway interactionsPatientsPatternProceduresProspective cohortProteinsProteomicsPsoriasisPublishingR-Series Research ProjectsR01 MechanismR01 ProgramRNARNA Gene ProductsRNA metabolismResearch GrantsResearch Project GrantsResearch ProjectsResearch ResourcesResourcesRheumatic DiseasesRheumatismRheumatologic DiseasesRheumatologic DisorderRibonucleic AcidSLESamplingSchilder-Addison ComplexSensitivity and SpecificitySiemerling-Creutzfeldt diseaseSiemerling-Creutzfeldt syndromeSignal TransductionSignal Transduction SystemsSignalingSiteSpottingsSymptomsSystemic Lupus ErythematosusSystemic Lupus ErythematousSystemic Lupus ErythmatosusSystemic diseaseTestingTimeTranscript Expression AnalysesTranscript Expression AnalysisValidationViral DiseasesVirus DiseasesX-Linked Adrenoleukodystrophyadrenocortical atrophy-cerebral sclerosis syndromeadrenoleukomyeloneuropathyanalyze gene expressionantibody assayantibody based testantibody testautoinflammatorybio-markersbiobankbiologic markerbiological signal transductionbiomarkerbiomarker signaturebiorepositorycandidate identificationcohortconstitutive expressionconstitutive gene expressiondevelopmentaldiagnostic biomarkerdiagnostic markerdisseminated lupus erythematosusearly detectionearly onsetenzyme linked immunoassayexperiencegene expression analysisgene expression assaygene testinggene-based testinggenetic testinghereditary disorderheritable disorderimmunogenimprovedimproved outcomeinborn errorinherited diseasesinherited genetic diseaseinherited genetic disorderinterestkidskinase inhibitorleukodystrophymRNAmelanodermic leukodystrophymicrocephaly-chorioretinopathy syndromenanostringneonatal periodneurotraumanewborn childnewborn childrennewborn screeningnoveloff-label applicationoff-label prescribingoff-label usepathwaypilot testpostnatalprospectiveprotein expressionpseudotoxoplasmosis syndromepsoriasiformpsoriaticresponsescreeningscreeningssexstandard of caresystemic lupus erythematosistranscriptional profilingvalidationsviral infectionvirus infectionvirus-induced diseaseyoungster
Description preview
Summary/Abstract for Project 2: Improved Presymptomatic Diagnosis in Aicardi Goutières Syndrome
Aicardi Goutières Syndrome (AGS) is an inborn error in RNA and DNA metabolism resulting in an early-onset
(neonatal or in first two years of life) leukodystrophy with extensive multisystemic disease and severe morbidity.
Off-label use of bariciticib, a…
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