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156 studies found for:    Spinal Muscular Atrophy

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Rank Status Study
1 Completed 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 Active, not recruiting Natural History of Spinal Muscular Atrophy Type 1 in Taiwan
Condition: Spinal Muscular Atrophy (SMA)
Intervention:
7 Completed Spinal Muscular Atrophy and Cardiac Autonomic Function
Condition: Spinal Muscular Atrophy
Intervention: Procedure: Air stacking manuever
8 Terminated Infants With Spinal Muscular Atrophy Type I
Condition: Spinal Muscular Atrophy
Intervention:
9 Active, not recruiting Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
Conditions: Type 2 Spinal Muscular Atrophy;   Type 3 Spinal Muscular Atrophy
Interventions: Other: Strength, function and activity measurements;   Other: Muscle MRI;   Other: Electrophysiology measurements;   Other: Blood sampling for biomarker analysis
10 Completed
Has Results 		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
11 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
12 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
13 Enrolling by invitation Mechanisms of Cell Death in Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention:
14 Active, not recruiting A Study to Assess the Efficacy and Safety of IONIS-SMN Rx in Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: IONIS-SMN Rx;   Procedure: Sham procedure
15 Completed Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproic Acid (VPA);   Drug: Placebo
16 Completed 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 Efficacy and Safety of IONIS-SMN Rx in Patients With Later-onset Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: IONIS-SMN Rx;   Procedure: Sham procedure
18 Completed
Has Results 		Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Sodium phenylbutyrate (NaPB)
19 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
20 Active, not recruiting A Study to Assess the Efficacy, Safety and Pharmacokinetics of IONIS SMNRx in Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: IONIS-SMNRx
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† Indicates status has not been verified in more than two years