Records containing: Spinal Muscular Atrophy (SMA)
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CLINICAL_TRIALS study record update · 2026-09-09A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2026-04-22A Phase 2 Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2026-04-21A Prospective, Longitudinal and Decentralised Study Investigating the Motor Development of Patients With Spinal Muscular Atrophy Identified by Newborn Screening Age 4 Years and Below: Active-NBS UK.
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CLINICAL_TRIALS study record update · 2026-04-16Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
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CLINICAL_TRIALS study record update · 2026-03-23Feasibility and Preliminary Effectiveness of HABIT-ILE + Functional Strength Training in Children With Spinal Muscular Atrophy (SMA): a Prospective Single-group Intervention Trial.
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CLINICAL_TRIALS study record update · 2026-03-13A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2026-01-22Effect of a Multicomponent Support Group on Self-Efficacy of Primary Caregivers of Children With Spinal Muscular Atrophy or Duchenne Muscular Dystrophy Enrolled in Treat-NMD Registry of Pakistan
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CLINICAL_TRIALS study record update · 2026-01-20Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
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CLINICAL_TRIALS study record update · 2026-01-20Observational Study for the Identification of Fatigue and Gait Biomarkers in Populations With Neuromuscular Pathologies in the Clinical Context and Daily Life
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CLINICAL_TRIALS study record update · 2026-01-16Understanding the Diagnostic Pathway and Treatment Experience of Patients With Spinal Muscular Atrophy (SMA)
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CLINICAL_TRIALS study record update · 2026-01-13Immediate and Sustained Effects of Intensive Neuroproprioceptive Equine-Assisted Physiotherapy in Children With Spinal Muscular Atrophy: A Randomized Crossover Trial
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CLINICAL_TRIALS study record update · 2026-01-12Detection of Cis Duplications of the SMN1 Gene Using Long-read Analysis to Address a Major Issue in Genetic Counseling for Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2026-01-07Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers
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CLINICAL_TRIALS study record update · 2025-12-16Active-NBS Liege - Monitoring the Motor Development of Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified Through Newborn Screening
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CLINICAL_TRIALS study record update · 2025-12-04Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
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CLINICAL_TRIALS study record update · 2025-12-03The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
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CLINICAL_TRIALS study record update · 2025-10-06Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions
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CLINICAL_TRIALS study record update · 2025-08-29Evaluation of a Portable Isokinetic Knee Training Device for Quadriceps Rehabilitation in Children With SMA
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CLINICAL_TRIALS study record update · 2025-07-18The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
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CLINICAL_TRIALS study record update · 2025-06-18Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2025-05-18Adult SMA REACH: Development and Implementation of a Standardised Data Set and Data Collection Research Study in the UK Adult SMA Population
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CLINICAL_TRIALS study record update · 2025-04-02A Randomized Crossover Pragmatic Study to Evaluate Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Patients With Spinal Muscular Atrophy. REALITY Study.
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CLINICAL_TRIALS study record update · 2025-03-21An Open-label, Dose-escalation Clinical Trial to Assess the Safety and Efficacy of EXG001-307 After Intrathecal Injection in Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2025-01-31A Prospective, Long-Term Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
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CLINICAL_TRIALS study record update · 2024-09-24Investigation of the Relationship Between Ultrasonographic Muscle Thickness Measurement and Echogenicity Assessment With Functional Scales in Patients With Spinal Muscular Atrophy: An Observational, Cross-Sectional, Case-Control Study