Records containing: Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2025-09-10Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
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CLINICAL_TRIALS study record update · 2025-08-26Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb Protocol
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CLINICAL_TRIALS study record update · 2025-07-22Study of AAV-hSMN1 (Vesemnogene Lantuparvovec) Gene Therapy in Subjects With Progressive Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2025-07-08A Registry-Based Parallel Group Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular Atrophy, With an Optional Open-Label Extension
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CLINICAL_TRIALS study record update · 2025-07-03A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
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CLINICAL_TRIALS study record update · 2025-06-03Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)
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CLINICAL_TRIALS study record update · 2025-05-28Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV
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CLINICAL_TRIALS study record update · 2025-05-25An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab
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CLINICAL_TRIALS study record update · 2025-05-06A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam
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CLINICAL_TRIALS study record update · 2025-04-29Motor NeuroProsthesis to Restore Motor Control for the COMMAND of Digital Devices: An Early Feasibility Study (EFS) of Safety in Subjects With Severe Quadriparesis
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CLINICAL_TRIALS study record update · 2025-04-09Myoelectric Devices for Restoration of Independent Arm Function in Children and Adults With Neurological Disease and Injury
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CLINICAL_TRIALS study record update · 2025-04-09An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2025-04-06French Register of Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2025-04-04Early Check: A Collaborative Innovation to Facilitate Pre-Symptomatic Clinical Trials in Newborns
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CLINICAL_TRIALS study record update · 2025-03-07Specificities of Quality of Life and Influence of Participation on the Quality of Life of the Adult with Spinal Muscular Atrophy in France: a Cross-sectional Study
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CLINICAL_TRIALS study record update · 2025-03-05A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 Spinal Muscular Atrophy (SMA)
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CLINICAL_TRIALS study record update · 2025-02-12Holter of Movement in Patients with Type 2 or 3 Spinal Muscular Atrophy When Initiating Treatment with Spinraza ° or Risdiplam. Acti-SMA Study.
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CLINICAL_TRIALS study record update · 2025-02-03A Monocentric, Prospective and Longitudinal Study Investigating the Acceptability, Feasibility, Safety and Efficacy of an Optimized Rehabilitation Program for Treated Patients With SMA Compared to the Current Rehabilitation Program in United Kingdom: ACE SMA.
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CLINICAL_TRIALS study record update · 2025-01-03Evaluation of Respiratory Functions, Thoracoabdominal Movements, and Exercise Capacity in Neuromuscular Diseases
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CLINICAL_TRIALS study record update · 2024-12-19Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
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CLINICAL_TRIALS study record update · 2024-12-04Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms): a Multicenter Single-case Experimental Design in Multiple Baselines Across Subjects, Randomized, Single-blinded Evaluation
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CLINICAL_TRIALS study record update · 2024-11-20A Prospective, Multi-center, Observational Study of the Safety, Tolerability and Effectiveness of SPINRAZA® (Nusinersen) in Adult Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-10-22An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443
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CLINICAL_TRIALS study record update · 2024-09-27Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural Environment
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CLINICAL_TRIALS study record update · 2024-09-03Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action