Muscular Atrophy - 50 Studies Found Active, not recruiting : A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) : Spinal Muscular Atrophy : 2013-04-22 : Drug: nusinersen Administered by intrathecal (IT) injection Completed : An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) : Spinal Muscular Atrophy : 2011-12-13 : Drug: nusinersen Administered by intrathecal (IT) injection Completed : A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) : Spinal Muscular Atrophy : 2014-11-12 : Drug: nusinersen Administered Completed : A Trial of Hydroxyurea in Spinal Muscular Atrophy : Spinal Muscular Atrophy : 2007-06-11 : Drug: Hydroxyurea Recruiting : Prospective Evaluation of Infants With Spinal Muscular Atrophy: : Spinal Muscular Atrophy : 2016-07-01 Terminated : A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) : Muscular Atrophy, Spinal : 2014-09-11 : Drug: RO6885247 oral solution Completed : A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (Firefish) : Muscular Atrophy, Spinal : 2016-09-21 : Drug: RO7034067 RO7034067 will be administered orally. Completed : A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish) : Muscular Atrophy, Spinal : 2016-09-19 : Drug: Placebo Placebo will be Completed : A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients : Muscular Atrophy, Spinal : 2007-12-04 : Drug: Hydroxyurea Completed : A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients : Muscular Atrophy, Spinal : 2007-12-04 : Drug: Hydroxyurea <<< Previous | Next >>>
