Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy [Phase 3]
This is an update on a Phase 3 trial evaluating Taldefgrobep Alfa for Spinal Muscular Atrophy, with the study currently active and not recru…
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CLINICAL_TRIALS research · 2026-01-30Clinical Trial Update: Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy [NA]
This clinical trial investigates new phenotypes among Spinal Muscular Atrophy patients treated with SMN-restoring therapy. The study is curr…
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CLINICAL_TRIALS research · 2026-01-22Clinical Trial Update: Self-Efficacy Enhancement Using a Multicomponent Support Group for Caregivers of Children With DMD/SMA [NA]
This clinical trial update describes a study to enhance self-efficacy in caregivers of children with Duchenne Muscular Dystrophy and Spinal …
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CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials [Phase 3]
This clinical trial update reports the long-term follow-up of patients with spinal muscular atrophy treated with OAV101 in Phase 3 trials.
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CLINICAL_TRIALS research · 2026-01-20Clinical Trial Update: Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases
This is a clinical trial update describing an observational study to assess variations in gait parameters in patients with neuromuscular dis…
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CLINICAL_TRIALS research · 2026-01-16Clinical Trial Update: Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System
This clinical trial update examines diagnostic journeys, patient experiences, and disparities in spinal muscular atrophy care within the Med…
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CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: Swiss Registry for Neuromuscular Disorders
The University of Bern is coordinating a Swiss registry for neuromuscular disorders with ongoing recruitment, covering multiple conditions i…
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CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) [Phase 3]
A Phase 3 clinical trial evaluating Salanersen (BIIB115) for movement effects and safety when given pre-symptomatically to genetically diagn…
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CLINICAL_TRIALS research · 2026-01-13Clinical Trial Update: Neuroproprioceptive Equine-Assisted Physiotherapy for Spinal Muscular Atrophy [NA]
A clinical trial update for an exploratory study on neuroproprioceptive equine-assisted physiotherapy for spinal muscular atrophy, currently…
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CLINICAL_TRIALS research · 2026-01-12Clinical Trial Update: Long Read Analysis in Spinal Muscular Atrophy - LOREASI [NA]
The LOREASI study is a clinical trial evaluating long-read analysis in spinal muscular atrophy, currently recruiting at University Hospital,…
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CLINICAL_TRIALS research · 2026-01-07Clinical Trial Update: Study of the Reproducibility of the French Version of the Modified SMAFRS Scale
This clinical trial evaluates the reproducibility of the French version of the Modified SMA Functional Assessment Scale (SMAFRS) in people w…
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CLINICAL_TRIALS research · 2026-01-07Clinical Trial Update: Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers [NA]
This clinical trial update describes the evaluation of a portable digital device for quantified analysis of markerless walking in volunteers…
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CLINICAL_TRIALS research · 2025-12-16Clinical Trial Update: Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening [NA]
An ongoing newborn-screening study evaluating decentralized monitoring of motor development in children with spinal muscular atrophy or Duch…
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CLINICAL_TRIALS research · 2025-12-04Clinical Trial Update: Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC). [Phase 3]
A Phase 3 real-world study is ongoing to evaluate Vesemnogene Lantuparvovec for spinal muscular atrophy in low- and middle-income countries.…
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CLINICAL_TRIALS research · 2025-12-03Clinical Trial Update: Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA® [NA]
This clinical trial update notes ongoing monitoring of motor function in adults with Spinal Muscular Atrophy Type II who are treated with Sp…
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CLINICAL_TRIALS research · 2025-11-21Clinical Trial Update: A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1 [NA]
This document reports a clinical study evaluating the safety and efficacy of SKG0201 Injection in patients with spinal muscular atrophy, cur…
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CLINICAL_TRIALS research · 2025-11-17Clinical Trial Update: CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain
A Spanish SMA observational study (NCT07231549) is actively recruiting to collect longitudinal data from patients with Spinal Muscular Atrop…
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CLINICAL_TRIALS research · 2025-11-04Clinical Trial Update: Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
This clinical trial update describes ongoing recruitment for a study assessing physiologic, pharmacodynamic, and clinical skeletal muscle re…
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CLINICAL_TRIALS research · 2025-10-06Clinical Trial Update: Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions
This clinical trial update outlines a planned psychological evaluation of how parents experience newborn screening for infantile Spinal Musc…
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CLINICAL_TRIALS research · 2025-09-15Clinical Trial Update: Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies [NA]
An ongoing study assessing motor function in infants with spinal muscular atrophy who are being treated with innovative therapies; the trial…
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CLINICAL_TRIALS research · 2025-09-15Clinical Trial Update: Pediatric Powered Wheelchair Standing Devices: An Exploratory Study [NA]
Exploratory study of pediatric powered wheelchair standing devices. The trial is currently active but not recruiting and focuses on children…
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CLINICAL_TRIALS research · 2025-09-03Clinical Trial Update: Pediatric Spinal Muscular Atrophy (SMA) China Registry
Biogen-sponsored pediatric spinal muscular atrophy registry in China is currently active and not recruiting.
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CLINICAL_TRIALS research · 2025-08-29Clinical Trial Update: Robot-assisted Training [NA]
A clinical trial conducted by Peking University Third Hospital is evaluating robot-assisted training for spinal muscular atrophy and is curr…
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CLINICAL_TRIALS research · 2025-08-26Clinical Trial Update: Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb [NA]
A clinical trial evaluating spinal cord stimulation to improve upper-limb motor function in spinal muscular atrophy is currently active but …
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CLINICAL_TRIALS research · 2025-07-22Clinical Trial Update: An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy [Phase 1, Phase 2]
Open-label Phase 1/2 trial evaluating Vesemnogene Lantuparvovec for spinal muscular atrophy; currently recruiting.