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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enrollment
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agemin
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PhaseCountries
1NCT03643562
(ClinicalTrials.gov)
June 18, 201819/8/2018Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous SystemOpen-label Evaluation of Adrabetadex in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)Niemann-Pick Type C DiseaseDrug: AdrabetadexMandos LLCNULLCompleted4 YearsN/AAll27Phase 3United States