grant
Project 1- Longitudinal Study
Organization BAYLOR COLLEGE OF MEDICINELocation HOUSTON, UNITED STATESPosted 15 Sept 2025Deadline 31 Aug 2030
NIHUS FederalResearch GrantFY20250-4 weeks old21+ years oldAcquired brain injuryAcuteAdultAdult HumanAgeAmino AcidsBloodBlood Reticuloendothelial SystemBrain InjuriesCardiacCatabolismClinicalClinical TreatmentClinical TrialsCobalaminComplicationCorpus StriatumCorpus striatum structureDataDefectDetectionDevelopmentDiagnosisDiagnosticDiseaseDisorderDrugsED visitER visitEmergency care visitEmergency department visitEmergency hospital visitEmergency room visitEmesisEventFrequenciesGenotypeGoalsGrafting ProcedureHepatic TransplantationHereditary Metabolic DisorderHospital AdmissionHospitalizationIQ DeficitInborn Errors of MetabolismIncidenceIndividualInjuryInterventionInvestigationKidneyKidney DiseasesKidney GraftingKidney TransplantationKidney TransplantsKidney Urinary SystemKnowledgeLifeLiver GraftingLiver TransplantLongitudinal StudiesMeasuresMedicationMetabolicNatural HistoryNeonatal ScreeningNephropathyNeurocognitiveNeurocognitive DeficitNeurologicNeurologicalNewborn InfantNewborn Infant ScreeningNewbornsNutritionalOrgan TransplantationOrgan TransplantsOutcomePancreatitisPatientsPharmaceutical PreparationsPhase 1/2 Clinical TrialPhase I/II Clinical TrialPhenotypePlayRecommendationRecurrenceRecurrentRenal DiseaseRenal GraftingRenal TransplantationRenal TransplantsResearchRisk FactorsRoleSeveritiesSiteSolidSpottingsStriate BodyStriatumTherapeuticVomitingadulthoodagesaminoacidbrain damagebrain-injuredclinical interventionclinical outcome assessmentclinical therapyclinical trial readinesscohortdevelopmentaldrug/agentearly childhoodfeedinggastrointestinalgene testinggene-based testinggenetic testingglutaric acidemiaglutaric aciduriaglutaricacidemiaimprovedinborn metabolism disorderinjuriesintelligence quotient deficitisovaleric acidemiaisovaleryl coA dehydrogenase deficiencyketotic glycinemiaketotic hyperglycinemiakidney disorderkidney txliver transplantationlong-term studylongitudinal outcome studiesmethylmalonic acidemiamethylmalonic aciduriamethylmalonyl coA mutase deficiencyneurocognitive declineneurocognitive impairmentnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynewborn childnewborn childrennewborn screeningnext generation therapeuticsnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapynutritiousorgan allograftorgan graftorgan xenograftpre-clinicalpre-clinical trialpreclinicalpreclinical trialpropionic acidemiapropionyl coA carboxylase deficiencyprospectiverenalrenal disorderresponseresponse to therapyresponse to treatmentsocial rolestriatalsuccesstherapeutic responsetherapy responsetreatment responsetreatment responsivenesstrial regimentrial treatment
Description preview
ABSTRACT – PROJECT 1 (LONGITUDINAL STUDY OF ORGANIC ACIDEMIAS)
In the past two decades, expanded newborn screening has transformed the diagnosis and management of
organic acidemias. Organic acidemias are inborn errors of metabolism that are due to enzymatic defects in amino
acid catabolism associated with life-threatening metabolic…
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