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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1NCT05682144
(ClinicalTrials.gov)
January 15, 202312/12/2022ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS IA Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and ScheieMucopolysaccharidosis IH/S;Mucopolysaccharidosis ISBiological: Autologous Plasmablasts (B cells)Immusoft CorporationNULLNot yet recruiting18 YearsN/AAll2Phase 1United States