Records containing: Muscular Atrophy, Spinal
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CLINICAL_TRIALS study results posted · 2026-10-30A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
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CLINICAL_TRIALS study record update · 2026-08-11A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
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CLINICAL_TRIALS study record update · 2026-04-13A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
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CLINICAL_TRIALS study record update · 2026-04-03A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2026-04-03A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
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CLINICAL_TRIALS study record update · 2026-03-20Real-World Evidence of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Analysis Based on REaDY Registry
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CLINICAL_TRIALS study record update · 2026-03-18An Open Label, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)
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CLINICAL_TRIALS study record update · 2026-03-09Spinraza (Nusinersen) SMA Pregnancy Exposure Study Within Existing SMA Registries
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CLINICAL_TRIALS study record update · 2026-03-03A Phase 3, Randomized, Double-Blinded Study to Evaluate the Safety and Efficacy of Salanersen (BIIB115) After Onasemnogene Abeparvovec Treatment in Infants With Genetically Diagnosed Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2026-02-18A Long-Term Extension Study of Nusinersen (BIIB058) Administered at Higher Doses in Participants With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
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CLINICAL_TRIALS study record update · 2026-02-11Disease-modifying Therapies (DMT) Treatment Outcomes in Patients With Spinal Muscular Atrophy (SMA) - A Real-world Study Using the Komodo Claims Data
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CLINICAL_TRIALS study record update · 2026-01-30Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy (SMA) Receiving Disease Modifying Therapies (DMT) - Real-world Analysis Using the Komodo Claims Data
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CLINICAL_TRIALS study record update · 2026-01-22Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy
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CLINICAL_TRIALS study record update · 2026-01-15An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2025-11-20Population-based New-Born Screening of Spinal Muscular Atrophy to Evaluate the Uptake and Feasibility in the UK Context
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CLINICAL_TRIALS study record update · 2025-10-14A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
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CLINICAL_TRIALS study record update · 2025-09-03Pediatric Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
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CLINICAL_TRIALS study record update · 2025-07-17A Multicenter, Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium) When Used in Routine Medical Practice in Korea
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CLINICAL_TRIALS study record update · 2025-07-11Adult Patients With Spinal Muscular Atrophy in China: A Nationwide Registry
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CLINICAL_TRIALS study record update · 2025-07-09A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study With Long-Term Extension in Pediatric SMA Participants Previously Treated With Onasemnogene Abeparvovec (Zolgensma™) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115
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CLINICAL_TRIALS study record update · 2025-06-05Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
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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 · 2025-05-02Examining the Effects of Trunk Control Training and Pulmonary Rehabilitation Program in Children With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-05-31A Phase IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerability and effIcacy of Gene Replacement Therapy With intravenousOAV101(AVXS101) in Pediatric Patients From Latin America With Spinal Muscular Atrophy (SMA) - OFELIA
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CLINICAL_TRIALS study record update · 2024-04-29A Brief Hope Intervention to Increase Hope Level and Decrease Stress Level of Parents With Children With Medical Complexity in the Community? A Pilot RCT