Status | Study |
Recruiting |
Study Name: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (Firefish) Condition: Muscular Atrophy, Spinal Date: 2016-09-21 Interventions: Drug: RO7034067 RO7034067 will be administered orally. |
Recruiting |
Study Name: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish) Condition: Muscular Atrophy, Spinal Date: 2016-09-19 Interventions: Drug: Placebo Placebo will be |
Active, not recruiting |
Study Name: A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Participants With Spinal Muscular Atrophy (SMA) Condition: Muscular Atrophy, Spinal Date: 2015-12-01 Interventions: Drug: Olesoxime Participants will receive homogeneous suspension of olesoxime. |
Active, not recruiting |
Study Name: A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). Condition: Spinal Muscular Atrophy Date: 2015-05-14 Interventions: Drug: Nusinersen Administered |
Active, not recruiting |
Study Name: An Open Label Study of LMI070 in Type 1 Spinal Muscular Atrophy (SMA) Condition: Spinal Muscular Atrophy Date: 2014-10-01 Interventions: Drug: LMI070 |
Terminated |
Study Name: A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) Condition: Muscular Atrophy, Spinal Date: 2014-09-11 Interventions: Drug: RO6885247 oral solution |
Completed |
Study Name: Newborn Screening for Spinal Muscular Atrophy Condition: Spinal Muscular Atrophy Date: 2014-04-21 Interventions: Other: newborn screening test for SMA Routine newborn screening dried blood spots sample is used to test |
Completed |
Study Name: Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers. Condition: Healthy Volunteer, Muscular Atrophy, Spinal Date: 2014-01-16 |
Completed |
Study Name: A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis Condition: Muscular Atrophy, Spinal Date: 2013-07-25 |
Enrolling by invitation |
Study Name: Mechanisms of Cell Death in Spinal Muscular Atrophy Condition: Spinal Muscular Atrophy Date: 2012-12-13 |