Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
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114 studies found for:    Spinal Muscular Atrophy

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Rank Status Study
1 Active, not recruiting Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Condition: Spinal Muscular Atrophy (SMA)
Intervention:
2 Completed A Pilot Study of Biomarkers for Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention:
3 Terminated
Has Results 		Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
Conditions: Spinal Muscular Atrophy Type II;   Spinal Muscular Atrophy Type III
Intervention: Drug: sodium phenylbutyrate
4 Active, not recruiting Newborn Screening for Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Other: newborn screening test for SMA
5 Terminated
Has Results 		Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
Condition: Spinal Muscular Atrophy Type I
Intervention: Drug: sodium phenylbutyrate
6 Completed Spinal Muscular Atrophy and Cardiac Autonomic Function
Condition: Spinal Muscular Atrophy
Intervention: Procedure: Air stacking manuever
7 Completed CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
Condition: Spinal Muscular Atrophy Type I
Intervention: Drug: Valproic Acid and Levocarnitine
8 Completed
Has Results 		Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproic Acid and Levocarnitine;   Drug: Placebo
9 Recruiting Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)
Condition: Infantile Spinal Muscular Atrophy of Type 2 or 3
Intervention: Other: Physical exercise in a swimming pool during 6 months
10 Enrolling by invitation Mechanisms of Cell Death in Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention:
11 Unknown † Infants With Spinal Muscular Atrophy Type I
Condition: Spinal Muscular Atrophy
Intervention:
12 Completed Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproic Acid (VPA);   Drug: Placebo
13 Recruiting A Study to Assess the Efficacy and Safety of ISIS-SMN Rx in Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: ISIS-SMN Rx;   Procedure: Sham procedure
14 Recruiting A Study to Assess the Efficacy and Safety of ISIS-SMN Rx in Patients With Later-onset Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: ISIS-SMN Rx;   Procedure: Sham procedure
15 Completed Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Sodium phenylbutyrate (NaPB)
16 Active, not recruiting Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
Conditions: Spinal Muscular Atrophy;   Neuromuscular Disease
Intervention: Other: Exercise
17 Active, not recruiting A Study to Assess the Safety and Pharmacokinetics of ISIS SMNRx in Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
18 Completed An Open-label Safety, Tolerability, and Dose-range Finding Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
19 Completed An Open-label Safety and Tolerability Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
20 Active, not recruiting An Open-label Safety, Tolerability and Dose-range Finding Study of Multiple Doses of ISIS SMNRx in Patient With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
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† Indicates status has not been verified in more than two years