Spinal Muscular Atrophy 1
Also known as: Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman] / Spinal Muscular Atrophy Type I / Infantile Spinal Muscular Atrophy / Infantile-onset Spinal Muscular Atrophy / Infantile-onset Spinal Muscular Atrophy (SMA) / Spinal muscular atrophy infantile onset / Infantile spinal muscular atrophy, type I [Werdnig-Hoffman] / Werdnig-Hoffmann disease / Werdnig-Hoffmann disease (disorder) / HMN (Hereditary Motor Neuropathy) Proximal Type I / Spinal muscular atrophy, unspecified / Muscular Atrophy, Spinal / Spinal amyotrophy / Spinal muscular atrophy (disorder) / Spinal muscular atrophy
Drug | Drug Name | Drug Description |
---|
Drug | Drug Name | Target | Type |
---|
Drug | Drug Name | Phase | Status | Count |
---|---|---|---|---|
DB02325 | Isopropyl alcohol | 1 | Completed | 1 |
DB15528 | Onasemnogene abeparvovec | 1 | Completed | 1 |
DB00583 | Levocarnitine | 1 / 2 | Completed | 1 |
DB06819 | Phenylbutyric acid | 1 / 2 | Terminated | 1 |
DB00313 | Valproic acid | 1 / 2 | Completed | 1 |
DB02325 | Isopropyl alcohol | 3 | Active Not Recruiting | 1 |
DB02325 | Isopropyl alcohol | 3 | Completed | 1 |
DB15528 | Onasemnogene abeparvovec | 3 | Active Not Recruiting | 1 |
DB15528 | Onasemnogene abeparvovec | 3 | Completed | 1 |
DB02325 | Isopropyl alcohol | Not Available | Active Not Recruiting | 1 |
DB13161 | Nusinersen | Not Available | No Longer Available | 1 |
DB15528 | Onasemnogene abeparvovec | Not Available | Active Not Recruiting | 1 |