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Results for “Ambulatory Spinal Muscular Atrophy”

13 trials

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Showing 13 of 13 results

Early research (Phase 1)Ended earlyNCT03381729
What this trial is testing

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
Novartis Gene Therapies 32
Large-scale testing (Phase 3)Study completedNCT01645787
What this trial is testing

Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients

Who this might be right for
Spinal Muscular Atrophy
Columbia University 11
Testing effectiveness (Phase 2)Looking for participantsNCT05794139
What this trial is testing

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular Atrophy
NMD Pharma A/S 54
Not applicableStudy completedNCT02341053
What this trial is testing

Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular DystrophyNeuromuscular Disability
Gillette Children's Specialty Healthcare 4
Testing effectiveness (Phase 2)Study completedNCT03781479
What this trial is testing

Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients

Who this might be right for
Muscular Atrophy, Spinal
Catalyst Pharmaceuticals, Inc. 13
Not applicableLooking for participantsNCT06839469
What this trial is testing

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

Who this might be right for
Spinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy (DMD)
Columbia University 106
Large-scale testing (Phase 3)Study completedNCT05089656
What this trial is testing

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Who this might be right for
Type 2 Spinal Muscular Atrophy
Novartis Pharmaceuticals 126
Not applicableLooking for participantsNCT07400198
What this trial is testing

Gait and Bone Health in SMA

Who this might be right for
Spinal Muscular Atrophy Type 3Ambulatory Spinal Muscular Atrophy
Jacqueline Montes 22
Large-scale testing (Phase 3)Active Not RecruitingNCT05337553
What this trial is testing

Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

Who this might be right for
Spinal Muscular AtrophyNeuromuscular DiseasesSMA
Biohaven Pharmaceuticals, Inc. 269
Not applicableStudy completedNCT02895789
What this trial is testing

Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

Who this might be right for
Spinal Muscular Atrophy Type 3Mitochondrial Myopathy
Columbia University 42
Not applicableStudy completedNCT04193085
What this trial is testing

Wearable Technology to Assess Gait Function in SMA and DMD

Who this might be right for
Spinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy
Columbia University 39
Not applicableStudy completedNCT02428673
What this trial is testing

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

Who this might be right for
OsteopeniaSpinal Muscular AtrophyCerebral Palsy+3 more
Gillette Children's Specialty Healthcare 6
Testing effectiveness (Phase 2)Ended earlyNCT03819660
What this trial is testing

Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3

Who this might be right for
Spinal Muscular Atrophy Type 3
Catalyst Pharmaceuticals, Inc. 13