19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details


臨床試験数 : 899 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

  
1 trial found
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1NCT01414985
(ClinicalTrials.gov)
April 15, 201013/12/2010AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid LipofuscinosisDirect CNS Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human CLN2 cDNA to Children With LINCL With Uncommon Genotypes and/or Moderate to Severe ImpairmentLate Infantile Neuronal Ceroid Lipofuscinosis;Batten DiseaseBiological: AAVrh.10CUCLN2Weill Medical College of Cornell UniversityNULLCompleted3 Years18 YearsAll8Phase 1/Phase 2United States