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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1NCT01299727
(ClinicalTrials.gov)
March 1, 201116/2/2011Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)An Open-Label Extension of Study HGT-SAN-055 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)Sanfilippo SyndromeBiological: rhHNS-10 mg;Biological: rhHNS-45 mg;Biological: rhHNS-90 mgShireNULLTerminated3 YearsN/AAll12Phase 1/Phase 2Netherlands;United Kingdom