Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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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 trial_result · 2025-12-23Clinical Trial Update: Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
This completed clinical trial study evaluated motor development and the use of orthoses in individuals with Spinal Muscular Atrophy.
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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 trial_result · 2025-12-10Clinical Trial Update: Implementation of FEES in Spinal Muscle Atrophy [NA]
This completed study reports on the implementation of FEES (Fiberoptic Endoscopic Evaluation of Swallowing) for patients with spinal muscula…
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CLINICAL_TRIALS trial_result · 2025-12-08Clinical Trial Update: Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) [Phase 3]
A Phase 3 trial evaluated the efficacy and safety of intrathecal AVXS-101 in pediatric patients with spinal muscular atrophy, with results p…
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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 trial_result · 2025-12-03Clinical Trial Update: Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy
A completed clinical trial update examining the mechanisms underlying exercise intolerance and persistent fatigue in spinal muscular atrophy…
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CLINICAL_TRIALS other · 2025-12-03Clinical Trial Update: The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder [NA]
A clinical trial evaluating a muscle-mimicking fabric-type shoulder orthosis to improve upper-limb movements in patients with neuromuscular …
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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 trial_result · 2025-11-20Clinical Trial Update: Newborn Screening for Spinal Muscular Atrophy
This update covers a newborn screening study for spinal muscular atrophy conducted by the University of Oxford, with the trial status shown …
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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 trial_result · 2025-10-20Clinical Trial Update: A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy [Phase 2]
Phase 2 study assessing multiple doses of nusinersen (ISIS 396443) delivered to presymptomatic infants with genetically diagnosed spinal mus…
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CLINICAL_TRIALS trial_result · 2025-10-14Clinical Trial Update: A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND) [Phase 4]
A Phase 4 clinical trial evaluating nusinersen in children with spinal muscular atrophy who previously received onasemnogene abeparvovec has…
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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 trial_result · 2025-10-01Clinical Trial Update: A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy [Phase 2]
Phase 2 results from a study of risdiplam in adult and pediatric participants with Spinal Muscular Atrophy have been completed and posted. T…