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156 studies found for:    Spinal Muscular Atrophy
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Rank Status Study
21 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
22 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
23 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
24 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
25 Completed Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
Condition: Spinal Muscular Atrophy
Intervention: Other: Functional tests
26 Not yet recruiting Oxidative Capacity and Exercise Tolerance in Ambulatory SMA
Conditions: Spinal Muscular Atrophy Type 3;   Mitochondrial Myopathy
Intervention:
27 Active, not recruiting Palliative Care in Spinal Muscular Atrophy (SMA) 1
Condition: Spinal Muscular Atrophy 1
Intervention: Other: Follow-up diary and questionnaire
28 Recruiting Prospective Evaluation of Infants With Spinal Muscular Atrophy:
Condition: Spinal Muscular Atrophy
Intervention:
29 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
30 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
31 Active, not recruiting Clinical Study of Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention:
32 Active, not recruiting Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
Condition: Spinal Muscular Atrophy 1
Intervention: Biological: AVXS-101
33 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
34 Completed Aerobic Training in Patients With Spinal Muscular Atrophy Type III
Condition: Spinal Muscular Atrophy
Intervention: Behavioral: Aerobic conditioning
35 Active, not recruiting
Has Results
Progressive Strength Training in Spinal Muscular Atrophy
Condition: Muscular Atrophy, Spinal
Intervention: Other: Progressive strength training
36 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
37 Completed Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid
Condition: Spinal Muscular Atrophy
Intervention:
38 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
39 Completed A Trial of Hydroxyurea in Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Hydroxyurea
40 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

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