19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 854 / 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04628871 (ClinicalTrials.gov) | November 3, 2020 | 9/11/2020 | Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX | Long-Term Follow-up of Subjects Who Were Treated With SB-318, SB-913, or SB-FIX, for Targeted Genome Editing Into the Albumin Gene in the Liver | Hemophilia B;Mucopolysaccharidosis I;Mucopolysaccharidosis II | Biological: SB-318;Biological: SB-913;Biological: SB-FIX | Sangamo Therapeutics | NULL | Enrolling by invitation | 18 Years | N/A | All | 13 | United States |