| Drug name |
Drug type |
Disease/phenotype |
Mechanism of action(MoA) |
Phase |
Status |
Source |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy | Survival motor neuron protein positive modulator | 3.0 | Completed | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | distal spinal muscular atrophy type 3 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | X-linked distal arthrogryposis multiplex congenita | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | Proximal spinal muscular atrophy type 2 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | Spinal muscular atrophy with respiratory distress type 1 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy | Survival motor neuron protein positive modulator | 3.0 | Not yet recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy, type 1 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | Distal hereditary motor neuropathy type 1 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | Proximal spinal muscular atrophy type 3 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | distal hereditary motor neuropathy type 2 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy | Survival motor neuron protein positive modulator | 1.0 | Completed | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy, type II | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy with respiratory distress type 2 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |