grant
Natural history of multisystem proteinopathy-1
Organization UNIVERSITY OF MIAMI SCHOOL OF MEDICINELocation CORAL GABLES, UNITED STATESPosted 30 Sept 2014Deadline 31 Aug 2030
NIHUS FederalResearch GrantFY202521+ years oldATP phosphohydrolaseATPaseAdenosine TriphosphataseAdultAdult HumanAdvocacyAgeAllelesAllelomorphsAmyotrophic Lateral SclerosisAmyotrophic Lateral Sclerosis Motor Neuron DiseaseBehavioralBiological MarkersBloodBlood PlasmaBlood Reticuloendothelial SystemBody SystemCell Cycle ControlCell Cycle RegulationCerebrospinal FluidCharacteristicsChildren's HospitalChromosome 9ClinicalClinical ResearchClinical StudyClinical TrialsClinical Trials DesignCognitiveCognitive DisturbanceCognitive ImpairmentCognitive declineCognitive function abnormalCollaborationsDNA Damage RepairDNA RepairDNA ReplicationDNA SynthesisDNA biosynthesisData CollectionDiseaseDisease ProgressionDisorderDisturbance in cognitionElectromyographyEnrollmentEnsureExtremitiesFTD dementiaFrequenciesFrontal Temporal DementiaFrontotemporal DementiaFutureGehrig's DiseaseGene ProteinsGene variantGenotypeGeographyHematopoietic Cell TumorHematopoietic MalignanciesHematopoietic NeoplasmsHematopoietic Neoplasms including LymphomasHematopoietic TumorHematopoietic and Lymphoid Cell NeoplasmHematopoietic and Lymphoid NeoplasmsHereditary DiseaseHeterogeneityHumanIBMPFDImmunoglobulin Enhancer-Binding ProteinImpaired cognitionInborn Genetic DiseasesInclusion Body Myopathy associated with Paget's disease of the boneInclusion Body Myopathy with Early-Onset Paget DiseaseInclusion body myopathy Paget’s disease of bone frontotemporal dementiaInclusion body myopathy associated with Paget’s disease of the bone and frontotemporal dementiaInclusion body myopathy with Paget’s disease of bone and frontotemporal dementiaIndustryInherited disorderIntracellular MembranesKnowledgeLightLimb structureLimbsLocationLou Gehrig DiseaseMalignant Hematopoietic NeoplasmMeasurementMeasuresMembrane FusionMetabolic Protein DegradationMethodsMissense MutationModern ManMotorMotor CellMotor NeuronsMulti-center studiesMulticenter StudiesMuscleMuscle TissueMuscular DystrophiesMyodystrophicaMyodystrophyNF-kBNF-kappa BNF-kappaBNFKBNatural HistoryNeurologicNeurologicalNeuromuscular conditionsNeuropsychologiesNeuropsychologyNon-TrunkNuclear Factor kappa BNuclear Transcription Factor NF-kBOrgan SystemOrphan DiseasePathogenicityPathologyPathway interactionsPatient RecruitmentsPatientsPediatric HospitalsPenetrancePerformancePhenotypePhotoradiationPlasmaPlasma SerumPre-Clinical ModelPreclinical ModelsPrevalenceProtein FamilyProtein Gene ProductsProtein TurnoverProteinsRare DiseasesRare DisorderRegulatory Protein DegradationResearch SpecimenReticuloendothelial System, Serum, PlasmaRiskSOD-1SOD-1 proteinSOD1SOD1 geneSOD1 gene productSiblingsSiteSite VisitSolidSourceSouth AfricaSpecimenSystemTestingTherapeuticTherapeutic EffectTrainingTranscription Factor NF-kBTravelTreatment EfficacyTumor TissueUnited KingdomUnited StatesUnscheduled DNA SynthesisUrineVCP diseaseVCP-associated diseaseVCP/p97Visitadulthoodagesallelic variantautosomebehavioral impairmentbio-markersbiobankbiologic markerbiomarkerbiomarker discoverybiomarker validationbiorepositoryblood cancercancer of bloodcancer of the bloodcerebral spinal fluidclinical careclinical outcome assessmentclinical trial readinesscognitive dysfunctioncognitive losscohortdecline in functiondecline in functional statusdisease causing variantdisease durationdisease heterogeneitydisease lengthdisease natural historydisease phenotypedisease-causing alleledisease-causing mutationefficacy outcomesenrollfront temporal dementiafrontal lobe dementiafrontotemporal lobar degeneration dementiafrontotemporal lobar dementiafrontotemporal lobe degeneration associated with dementiafunctional declinefunctional status declinegene editing platformgene editing systemgene editing technologygene editing toolsgene-editing toolkitgenetic variantgenomic varianthereditary disorderheritable disorderillness lengthimpaired behaviorimpressioninborn errorinherited diseasesinherited genetic diseaseinherited genetic disorderintervention efficacykappa B Enhancer Binding Proteinknock-downknockdownmarker validationmembermissense single nucleotide polymorphismmissense single nucleotide variantmissense variantmotoneuronmotor diseasemotor disordermotor dysfunctionmouse modelmultisystem proteinopathymurine modelmuscle dystrophymuscularneurofilamentneuropsychologicnuclear factor kappa betaorphan disorderp97-VCP proteinparticipant recruitmentpathogenic allelepathogenic variantpathwaypre-clinical studypreclinical studyprimary outcomeprospectiveprotein degradationremote assessmentremote evaluationsmall molecular inhibitorsmall molecule inhibitorspinal fluidsuperoxide dismutase 1therapeutic agent developmenttherapeutic developmenttherapeutic efficacytherapeutic targettherapy efficacytrial planningvalosin containing protein diseasevalosin containing protein p97valosin-containing proteinvalosine-containing protein
Description preview
PROJECT SUMMARY (Project 4: Natural History of MSP1)
This proposal’s primary objective is to validate a promising measure of motor function as a future efficacy out-
come in a large cohort of patients with multisystem proteinopathy 1 (MSP1) due to a pathogenic missense vari-
ant in the valosin-containing protein (VCP) gene. Secondarily, we will…
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