grant
Pre-Symptomatic Familial ALS (Pre-fALS) Study: From Prodrome and Biomarkers to ALS/FTD Prevention
Organization UNIVERSITY OF MIAMI SCHOOL OF MEDICINELocation CORAL GABLES, UNITED STATESPosted 15 Sept 2018Deadline 31 Jul 2029
NIHUS FederalResearch GrantFY2025Active Follow-upAmyotrophic Lateral SclerosisAmyotrophic Lateral Sclerosis Motor Neuron DiseaseAmyotrophic lateral sclerosis and frontotemporal degenerationAmyotrophic lateral sclerosis and frontotemporal dementiaBasal MetabolismBasal metabolic rateBehavioralBiochemicalBiologicalBiological MarkersBloodBlood Reticuloendothelial SystemBody CompositionC9ORF72ClinicalClinical MarkersCognitiveCollaborationsCox Proportional Hazards ModelsDataDegenerative Neurologic DisordersDevelopmentDiagnosisDifferences between sexesDiffers between sexesDiseaseDisease MarkerDisorderEarly InterventionEnrollmentEvaluationEventFTD dementiaFTD/ALSFTLD/ALSFrontal Temporal DementiaFrontotemporal DementiaFrontotemporal Lobar Degeneration/Amyotrophic lateral sclerosisFundingGehrig's DiseaseGenetic RiskGenotypeGoalsGrantImpairmentIndividualKnowledgeLightLou Gehrig DiseaseMarker DiscoveryMeasurementMetabolicMethodsModelingMotorNatural HistoryNervous System Degenerative DiseasesNeural Degenerative DiseasesNeural degenerative DisordersNeurodegenerative DiseasesNeurodegenerative DisordersNeurologic Degenerative ConditionsPathogenicityPathologyPatientsPersonsPhasePhenotypePhotoradiationPopulationPopulation GeneticsPredispositionPreventionPrevention trialProgressive DiseaseProspective StudiesResearch ResourcesResearch SpecimenResourcesRiskSOD-1SOD-1 proteinSOD1SOD1 geneSOD1 gene productSex DifferencesSexual differencesSkeletal MuscleSpecimenSubgroupSusceptibilitySyndromeTAR DNA binding protein 43 kDa pathologyTAR DNA binding protein 43 pathologyTAR DNA binding protein of 43 proteinopathyTDP43 associated neurodegenerationTDP43 associated neurodegenerative diseaseTDP43 associated pathologiesTDP43 induced neurodegenerationTDP43 neurodegenerationTDP43 neurodegenerative diseaseTDP43 neuropathologyTDP43 pathogenesisTDP43 pathologyTDP43 proteinopathyTDP43 related neurodegenerationTDP43 related pathologyTherapeuticTimeTrans active response DNA binding protein 43 pathologyTrans active response DNA binding protein of 43 kDa proteinopathyTranslationsTreesVariantVariationVoluntary MuscleWorkactive followupamyotrophic lateral sclerosis with frontotemporal dementiaamyotrophic lateral sclerosis/FTLDamyotrophic lateral sclerosis/frontotemporal dementiaamyotrophic lateral sclerosis/ftdanalyzing longitudinalbehavioral impairmentbio-markersbiologicbiologic markerbiomarkerbiomarker discoverychromosome 9 open reading frame 72clinical biomarkersclinically useful biomarkerscohortdegenerative diseases of motor and sensory neuronsdegenerative neurological diseasesdesigndesigningdevelopmentaldiagnostic criteriadisease causing variantdisease preventiondisease riskdisease-causing alleledisease-causing mutationdisorder preventiondisorder riskempowermentenrollfALSfamilial ALSfamilial amyotrophic lateral sclerosisfollow upfollow-upfollowed upfollowupfront temporal dementiafrontal lobe dementiafrontotemporal dementia-amyotrophic lateral sclerosisfrontotemporal lobar degeneration dementiafrontotemporal lobar dementiafrontotemporal lobar dementia amyotrophic lateral sclerosisfrontotemporal lobe degeneration associated with dementiaimpaired behaviorindexingindustrial partnershipindustry partnerindustry partnershiplongitudinal analysismild cognitive disordermild cognitive impairmentmotor impairmentmovement impairmentmovement limitationmulti-modalitymultimodalityneurodegenerative illnessneurofilamentnovelpathogenic allelepathogenic variantphenotypic datapredictive biological markerpredictive biomarkerspredictive markerpredictive molecular biomarkerpreventpreventingresting metabolic ratesex based differencessex-dependent differencessex-related differencessex-specific differencessuccesssuperoxide dismutase 1therapeutic agent developmenttherapeutic developmenttrans active response DNA binding protein 43 kDa pathologytrans active response DNA binding protein 43 proteinopathytranslation
Description preview
PROJECT SUMMARY
Amyotrophic lateral sclerosis (ALS) remains a fatal neurodegenerative disorder, in part because diagnosis is
delayed and treatment is initiated late in disease course. Therapeutic success is most likely when treatment is
initiated early, ideally while still pre-symptomatic. Unaffected carriers of pathogenic variants associated with…
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