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84 studies found for:    "spinal muscular atrophy"
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Rank Status Study
21 Completed Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
Condition: Spinal Muscular Atrophy (SMA)
Intervention:
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 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
24 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
25 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
26 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
27 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
28 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
29 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
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 Completed A Trial of Hydroxyurea in Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Hydroxyurea
32 Recruiting Prospective Evaluation of Infants With Spinal Muscular Atrophy:
Condition: Spinal Muscular Atrophy
Intervention:
33 Recruiting Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproate, Levocarnitine;   Drug: Placebo
34 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
35 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
36 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
37 Active, not recruiting Natural History of Spinal Muscular Atrophy Type 1 in Taiwan
Condition: Spinal Muscular Atrophy (SMA)
Intervention:
38 Not yet recruiting Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy (SMA)
Intervention: Drug: celecoxib
39 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:
40 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

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