| RISDIPLAM | Small molecule | none | SMN2 pre-mRNA positive modulator | 4 | ['Not yet recruiting', 'Recruiting', 'Completed', 'Active, not recruiting', 'Recruiting', '-', '-', 'Recruiting'] | ['ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'FDA', 'DailyMed', 'ClinicalTrials'] |
| ONASEMNOGENE ABEPARVOVEC | Gene | none | Survival motor neuron protein positive modulator | 3 | ['Active, not recruiting', 'Completed', 'Not yet recruiting', 'Active, not recruiting', 'Active, not recruiting', 'Active, not recruiting', 'Active, not recruiting', 'Active, not recruiting', 'Active, not recruiting', 'Active, not recruiting', 'Active, not recruiting', 'Active, not recruiting', 'Completed'] | ['ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials'] |
| NUSINERSEN | Oligonucleotide | none | SMN2 pre-mRNA positive modulator | 4 | ['Recruiting', 'Terminated', 'Enrolling by invitation', 'Recruiting', 'Terminated', 'Completed', 'Completed', 'Recruiting', 'Completed', 'Active, not recruiting', 'Active, not recruiting'] | ['ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials', 'ClinicalTrials'] |
| NUSINERSEN SODIUM | Oligonucleotide | none | SMN2 pre-mRNA positive modulator | 4 | ['-'] | ['FDA'] |