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
| 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 | NCT03204370 (ClinicalTrials.gov) | February 1, 2018 | 7/6/2017 | Natural History of Atypical Morquio A Disease | Natural History of Atypical Morquio A Disease: a 5-years Prospective Study in a Series of 9 Adult Patients Followed in a Single Expert Center | Mucopolysaccharidosis IV A | Drug: Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM] | GOIZET | BioMarin Pharmaceutical | Active, not recruiting | 18 Years | N/A | All | 10 | France |