Status | Study |
Completed |
Study Name: Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development Condition: Spinal Muscular Atrophy (SMA) Date: 2012-11-19 |
Completed |
Study Name: An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Condition: Spinal Muscular Atrophy Date: 2012-10-08 Interventions: Drug: Nusinersen Single IT injection for each dose O |
Active, not recruiting |
Study Name: Palliative Care in Spinal Muscular Atrophy (SMA) 1 Condition: Spinal Muscular Atrophy 1 Date: 2012-07-20 Interventions: Other: Follow-up diary and questionnaire A follow-up diary will be completed by the families and the dif |
Completed |
Study Name: Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe) Condition: Spinal Muscular Atrophy Date: 2012-05-30 Interventions: Other: Functional tests |
Terminated |
Study Name: Infants With Spinal Muscular Atrophy Type I Condition: Spinal Muscular Atrophy Date: 2012-03-06 |
Completed |
Study Name: Spinal Muscular Atrophy and Cardiac Autonomic Function Condition: Spinal Muscular Atrophy Date: 2012-01-24 Interventions: Procedure: Air stacking manuever Electrocardiogram signals were recorded for analyses of heart rate vari |
Completed |
Study Name: An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Condition: Spinal Muscular Atrophy Date: 2011-12-13 Interventions: Drug: nusinersen Administered by intrathecal (IT) injection |
Active, not recruiting |
Study Name: Progressive Strength Training in Spinal Muscular Atrophy Condition: Muscular Atrophy, Spinal Date: 2010-10-19 Interventions: Other: Progressive strength training the systematic increase in resistance weights |
Completed |
Study Name: A Pilot Study of Biomarkers for Spinal Muscular Atrophy Condition: Spinal Muscular Atrophy Date: 2008-09-18 |
Completed |
Study Name: Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy Condition: Spinal Muscular Atrophy Date: 2007-09-10 Interventions: Drug: Sodium phenylbutyrate (NaPB) The powder form of the drug will be dispensed. The target NaPB dosing |