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87 studies found for:    spinal muscular atrophy | "Spinal muscular atrophy"
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Rank Status Study
21 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
22 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
23 Recruiting Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
Condition: Spinal Muscular Atrophy Type 3
Intervention: Drug: Pyridostigmine Bromide
24 Recruiting Prospective Evaluation of Infants With Spinal Muscular Atrophy:
Condition: Spinal Muscular Atrophy
Intervention:
25 Completed An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
Condition: Spinal Muscular Atrophy
Intervention: Drug: nusinersen
26 Enrolling by invitation A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies.
Condition: Spinal Muscular Atrophy
Intervention: Drug: nusinersen
27 Completed
Has Results
An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Nusinersen
28 Completed An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
Condition: Spinal Muscular Atrophy
Intervention: Drug: nusinersen
29 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
30 Active, not recruiting A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
Condition: Spinal Muscular Atrophy
Intervention: Drug: nusinersen
31 Completed A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
Condition: Spinal Muscular Atrophy
Interventions: Drug: nusinersen;   Procedure: Sham procedure
32 Active, not recruiting A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
Condition: Spinal Muscular Atrophy
Interventions: Drug: Nusinersen;   Procedure: Sham Procedure
33 Completed An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
Condition: Spinal Muscular Atrophy
Intervention: Drug: nusinersen
34 Completed A Trial of Hydroxyurea in Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Hydroxyurea
35 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
36 Recruiting A Study of RO7034067 in Adult and Pediatric Participants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: RO7034067
37 Recruiting Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproate, Levocarnitine;   Drug: Placebo
38 Active, not recruiting A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Nusinersen
39 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
Intervention: Drug: nusinersen
40 Completed A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: nusinersen;   Procedure: Sham procedure

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Study has passed its completion date and status has not been verified in more than two years.