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83 studies found for:    "spinal muscular atrophy"
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Rank Status Study
21 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)
22 Enrolling by invitation An Open-Label Study (SHINE) for Patients With Spinal Muscular Atrophy (SMA) Who Participated in Studies With IONIS-SMNRx
Condition: Spinal Muscular Atrophy
Intervention: Drug: IONIS-SMN Rx
23 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
24 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
25 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
26 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
27 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
28 Completed 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
29 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
30 Active, not recruiting An Open-label Safety and Tolerability Study of IONIS SMNRx in Patients With Spinal Muscular Atrophy Who Previously Participated in IONIS SMNRx-CS2 or IONIS SMNRx-CS10
Condition: Spinal Muscular Atrophy
Intervention: Drug: IONIS-SMNRx
31 Active, not recruiting A Study to Assess the Safety and Tolerability of ISIS 396443 in Participants With Spinal Muscular Atrophy (SMA).
Condition: Spinal Muscular Atrophy
Interventions: Drug: ISIS 396443;   Procedure: Sham Procedure
32 Not yet recruiting Oxidative Capacity and Exercise Tolerance in Ambulatory SMA
Conditions: Spinal Muscular Atrophy Type 3;   Mitochondrial Myopathy
Intervention:
33 Temporarily not available Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
Condition: Infantile-onset Spinal Muscular Atrophy (SMA)
Intervention: Drug: nusinersen
34 Active, not recruiting Clinical Study of Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention:
35 Active, not recruiting Natural History of Spinal Muscular Atrophy Type 1 in Taiwan
Condition: Spinal Muscular Atrophy (SMA)
Intervention:
36 Recruiting Prospective Evaluation of Infants With Spinal Muscular Atrophy:
Condition: Spinal Muscular Atrophy
Intervention:
37 Completed A Trial of Hydroxyurea in Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Hydroxyurea
38 Recruiting Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproate, Levocarnitine;   Drug: Placebo
39 Recruiting A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: CK-2127107 150 mg;   Drug: Placebo;   Drug: CK-2127107 450 mg
40 Not yet recruiting Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy (SMA)
Intervention: Drug: celecoxib

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Indicates status has not been verified in more than two years