| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy, type II | 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 |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 2.0 | Completed | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy | Survival motor neuron protein positive modulator | 1.0 | Completed | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy | Survival motor neuron protein positive modulator | 3.0 | Completed | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy with respiratory distress type 2 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| NUSINERSEN SODIUM | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 4.0 | - | FDA |
| RISDIPLAM | Small molecule | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 4.0 | Recruiting | ClinicalTrials |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 2.0 | 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 |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 3.0 | Enrolling by invitation | 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 | distal spinal muscular atrophy type 3 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 2.0 | Terminated | ClinicalTrials |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 2.0 | Recruiting | ClinicalTrials |
| RISDIPLAM | Small molecule | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 2.0 | Recruiting | ClinicalTrials |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 1.0 | Completed | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | Proximal spinal muscular atrophy type 3 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 3.0 | Recruiting | ClinicalTrials |
| RISDIPLAM | Small molecule | Atrophy | SMN2 pre-mRNA positive modulator | 4.0 | - | DailyMed |
| RISDIPLAM | Small molecule | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 2.0 | Active, not recruiting | ClinicalTrials |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 3.0 | Terminated | ClinicalTrials |
| RISDIPLAM | Small molecule | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 4.0 | - | FDA |
| ONASEMNOGENE ABEPARVOVEC | Gene | X-linked distal arthrogryposis multiplex congenita | 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 |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 4.0 | Recruiting | ClinicalTrials |
| NUSINERSEN | Oligonucleotide | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 3.0 | Completed | ClinicalTrials |
| RISDIPLAM | Small molecule | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 4.0 | Not yet recruiting | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | Proximal spinal muscular atrophy type 2 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| RISDIPLAM | Small molecule | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 1.0 | Completed | ClinicalTrials |
| ONASEMNOGENE ABEPARVOVEC | Gene | spinal muscular atrophy, type 1 | Survival motor neuron protein positive modulator | 3.0 | Active, not recruiting | ClinicalTrials |
| RISDIPLAM | Small molecule | spinal muscular atrophy | SMN2 pre-mRNA positive modulator | 1.0 | Recruiting | ClinicalTrials |