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43 studies found for:    "Spinal muscular atrophy"

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Rank Status Study
1 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
2 Recruiting Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
Condition: Spinal Muscular Atrophy
Intervention: Other: Functional tests
3 Recruiting Infants With Spinal Muscular Atrophy Type I
Condition: Spinal Muscular Atrophy
Intervention:
4 Active, not recruiting Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
Conditions: Spinal Muscular Atrophy Type II;   Spinal Muscular Atrophy Type III Non Ambulant
Interventions: Drug: Olesoxime;   Drug: Placebo
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 Recruiting Palliative Care in Spinal Muscular Atrophy (SMA) 1
Condition: Spinal Muscular Atrophy 1
Intervention: Other: Follow-up diary and questionnaire
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 A Pilot Study of Biomarkers for Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention:
9 Enrolling by invitation Mechanisms of Cell Death in Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention:
10 Completed Spinal Muscular Atrophy and Cardiac Autonomic Function
Condition: Spinal Muscular Atrophy
Intervention: Procedure: Air stacking manuever
11 Active, not recruiting Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Sodium phenylbutyrate (NaPB)
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 Safety and Pharmacokinetics of ISIS SMNRx in Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
14 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
15 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
16 Completed Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Valproic Acid
17 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
18 Recruiting Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
Conditions: Spinal Muscular Atrophy;   Neuromuscular Disease
Intervention: Other: Exercise
19 Recruiting 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 Recruiting Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Condition: Spinal Muscular Atrophy (SMA)
Intervention:
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† Indicates status has not been verified in more than two years