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

DrugDrug NameDrug Description
DrugDrug NameTargetType
DrugDrug NamePhaseStatusCount
DB02325Isopropyl alcohol1Completed1
DB15528Onasemnogene abeparvovec1Completed1
DB00583Levocarnitine1 / 2Completed1
DB06819Phenylbutyric acid1 / 2Terminated1
DB00313Valproic acid1 / 2Completed1
DB02325Isopropyl alcohol3Active Not Recruiting1
DB02325Isopropyl alcohol3Completed1
DB15528Onasemnogene abeparvovec3Active Not Recruiting1
DB15528Onasemnogene abeparvovec3Completed1
DB02325Isopropyl alcoholNot AvailableActive Not Recruiting1
DB13161NusinersenNot AvailableNo Longer Available1
DB15528Onasemnogene abeparvovecNot AvailableActive Not Recruiting1