Spinal Muscular Atrophy
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CLINICAL_TRIALS trial_result · 2026-08-11Clinical Trial Update: A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD) [Phase 3]
A phase 3 trial update assessing long-term safety of higher doses of nusinersen (BIIB058) in SMA participants from a prior ONWARD trial.
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CLINICAL_TRIALS trial_result · 2026-04-23Clinical Trial Update: Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback [NA]
A completed clinical trial assessed the feasibility of virtual reality in children with neuromuscular diseases and evaluated the effectivene…
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CLINICAL_TRIALS trial_result · 2026-04-20Clinical Trial Update: Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) [Phase 4]
Phase 4 long-term follow-up study of risdiplam in Spinal Muscular Atrophy; the trial is currently active but not recruiting.
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CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders [NA]
A terminated clinical trial update investigating gut peptides and bone remodeling in children with neuromuscular disorders, sponsored by the…
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CLINICAL_TRIALS trial_result · 2026-04-15Clinical Trial Update: A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
A Roche-sponsored clinical trial evaluating risdiplam for spinal muscular atrophy types I and II is listed as NOT_YET_RECRUITING.
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy [Phase 2]
Phase 2 study evaluating the safety and efficacy of NMD670 in ambulatory adult patients with Type 3 spinal muscular atrophy; the trial is sp…
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CLINICAL_TRIALS trial_result · 2026-03-18Clinical Trial Update: ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM) [Phase 4]
A U.S. pragmatic Phase 4 study (STREAM) is evaluating real-world safety and effectiveness of intrathecal onasemnogene abeparvovec-brve (OAV1…
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CLINICAL_TRIALS trial_result · 2026-03-16Clinical Trial Update: Spinal Cord Stimulation in Spinal Muscular Atrophy [NA]
A completed clinical trial evaluating a spinal cord stimulator in patients with Spinal Muscular Atrophy (Types 3 and 4) has posted results a…
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CLINICAL_TRIALS trial_result · 2026-02-23Clinical Trial Update: Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
A completed clinical trial studying neuromuscular junction defects in spinal muscular atrophy following restoration of SMN protein in centra…
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CLINICAL_TRIALS trial_result · 2026-02-23Clinical Trial Update: Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)
This clinical trial update reports the completion of a study assessing Motor Unit Number Estimation (MUNE) in adults with Spinal Muscular At…
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CLINICAL_TRIALS trial_result · 2026-02-11Clinical Trial Update: A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
This completed real-world clinical trial evaluates outcomes of disease-modifying therapy for patients with spinal muscular atrophy.
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CLINICAL_TRIALS trial_result · 2026-01-30Clinical Trial Update: A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
A completed clinical trial examining dosing patterns and costs in spinal muscular atrophy patients receiving disease-modifying therapies.
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CLINICAL_TRIALS trial_result · 2026-01-22Clinical Trial Update: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam [Phase 3]
Phase 3 trial update evaluating the efficacy and safety of Apitegromab in patients with later-onset spinal muscular atrophy who are treated …
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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 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 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 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 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 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…
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CLINICAL_TRIALS trial_result · 2025-09-11Clinical Trial Update: Palliative Care in Spinal Muscular Atrophy (SMA) 1 [NA]
This clinical trial update notes the completion of a study on palliative care in Spinal Muscular Atrophy type 1.
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CLINICAL_TRIALS trial_result · 2025-06-05Clinical Trial Update: Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy [Phase 3]
Phase 3 clinical trial results for nusinersen (BIIB058) in spinal muscular atrophy have been completed. The results were posted on 2025-06-0…
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CLINICAL_TRIALS trial_result · 2025-06-03Clinical Trial Update: Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam [Phase 3]
Phase IIIb, open-label, multicenter study evaluating the safety, tolerability, and efficacy of intrathecal OAV101 in spinal muscular atrophy…
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CLINICAL_TRIALS trial_result · 2025-05-28Clinical Trial Update: Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV [NA]
An exploratory study evaluating respiratory bacterial infections, superinfections, and colonizations in spinal muscular atrophy patients usi…