AAV2CUhCLN2 (3x10^12 particle units) ( DrugBank: - )


1 disease
IDDisease name (Link within this page)Number of trials
19Lysosomal storage disease1

19. Lysosomal storage disease


Clinical trials : 899 Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
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agemin
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PhaseCountries
1NCT00151216
(ClinicalTrials.gov)
June 20046/9/2005Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid LipofuscinosisAdministration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children With Late Infantile Neuronal Ceroid LipofuscinosisBatten Disease;Late Infantile Neuronal Ceroid LipofuscinosisBiological: AAV2CUhCLN2 (3x10^12 particle units)Weill Medical College of Cornell UniversityNathan's Battle FoundationCompleted3 Years18 YearsAll10Phase 1United States