grant
Batten Disease Clinical Research Consortium
Organization HUGO W. MOSER RES INST KENNEDY KRIEGERLocation BALTIMORE, UNITED STATESPosted 15 Sept 2025Deadline 31 Aug 2030
NIHUS FederalResearch GrantFY2025Active Follow-upAddressAdvanced DevelopmentAffectAgeAmentiaBatten DiseaseBatten-Mayou DiseaseBatten-Spielmeyer-Vogt DiseaseBlindnessCLN3CLN3 geneCLN3 proteinCLN5CLN5 geneCLN5 proteinCLN6CLN6 geneCLN6 proteinCaringChildhoodClinicalClinical ResearchClinical StudyClinical TrialsClinical Trials DesignCollaborationsCommunitiesDNA TherapyDegenerative Neurologic DisordersDementiaDiseaseDisorderDrug TherapyEnsureEpilepsyEpileptic SeizuresEpilepticsEvaluationFoundationsFutureGene Transfer ClinicalGene therapy trialGenesGenetic InterventionIndividualJuvenile Cerebroretinal DegenerationKnowledgeMeasurementMentorsMethodsMissionNatural HistoryNerve DegenerationNervous System Degenerative DiseasesNeural Degenerative DiseasesNeural degenerative DisordersNeurodegenerative DiseasesNeurodegenerative DisordersNeurologic Degenerative ConditionsNeuron DegenerationNeuronal Ceroid-LipofuscinosisOnset of illnessOrphan DiseaseOutcome MeasurePathogenicityPathologyPharmacological TreatmentPharmacotherapyPilot ProjectsR-Series Research ProjectsR01 MechanismR01 ProgramRare DiseasesRare DisorderReproducibilityResearchResearch GrantsResearch Project GrantsResearch ProjectsResearch ResourcesResourcesSamplingScienceScientific Advances and AccomplishmentsScientistSeizure DisorderServicesSpielmeyer-Vogt DiseaseStructureSymptom BurdenSymptomsSyndromeTechnologyTestingTherapeuticTrainingTranslational ResearchTranslational Research EnterpriseTranslational ScienceTranslationsVariantVariationWorkactive followupadvocacy organizationsagesbiobankbiorepositorycareerceroid lipofuscinosisclinical research siteclinical siteclinical trial implementationcommunity partnershipdata qualitydegenerative diseases of motor and sensory neuronsdegenerative neurological diseasesdesigndesigningdiscover genesdisease onsetdisorder onsetdrug interventiondrug treatmentepilepsiaepileptogenicfollow upfollow-upfollowed upfollowupgene discoverygene repair therapygene therapygene transfer trialgene-based therapygene-based treatmentgene-directed therapygene-targeted therapygene-targeted treatmentgenetic therapygenomic therapyhereditary ceroid lipofuscinosisimplementation of clinical trialsimprovedlipopigmentsmeasurable outcomemotor diseasemotor disordermotor dysfunctionnerve cell deathnerve cell lossneural degenerationneurodegenerationneurodegenerativeneurodegenerative illnessneurological degenerationneuron cell deathneuron cell lossneuron deathneuron lossneuronal 5 ceroid-lipofuscinosisneuronal cell deathneuronal cell lossneuronal deathneuronal degenerationneuronal lossnew drug treatmentsnew drugsnew pharmacological therapeuticnew therapeuticsnew therapynext generationnext generation therapeuticsnovelnovel drug treatmentsnovel drugsnovel pharmaco-therapeuticnovel pharmacological therapeuticnovel therapeuticsnovel therapyorphan disorderoutcome measurementpediatricpharmaceutical interventionpharmacological interventionpharmacological therapypharmacology interventionpharmacology treatmentpharmacotherapeuticspilot studypre-clinicalpreclinicalprogramsscientific accomplishmentsscientific advancessmall moleculetooltranslationtranslation researchtranslation research enterprisetranslational investigationtranslational research programtrial readinessvision lossvisual loss
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CAREER ENHANCEMENT CORE – PROJECT SUMMARY/ABSTRACT
The neuronal ceroid lipofuscinoses (NCLs, Batten disease) are collectively the most prevalent childhood neurodegenerative diseases. Despite a growing understanding of the molecular and cellular pathophysiology, too few clinician investigators are conducting NCL translational research. This…
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