Records containing: SMA
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-03-25A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)
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CLINICAL_TRIALS study record update · 2026-02-04A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension
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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 · 2026-01-15Swiss Registry for Neuromuscular Disorders (Swiss-Reg-NMD)
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CLINICAL_TRIALS study record update · 2026-01-07Study of the Reproducibility of the French Version of the Modified SMAFRS Scale
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CLINICAL_TRIALS study record update · 2025-08-26Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb Protocol
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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 · 2025-04-25A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
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CLINICAL_TRIALS study record update · 2025-03-14Expanded Access Protocol of Apitegromab for Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2024-07-23Spinal Muscular Atrophy Patient Registry of the United Kingdom and Ireland
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CLINICAL_TRIALS study record update · 2024-03-21A Head-to-head Study Comparing the Functional Value of Two Models of Robotically Assisted Rehabilitation in SMA Patients: Single-center, Randomized, Single-blinded, Comparative Study of a Robotically Active Verticalization Model Versus Robotically Assisted Locomotion Model
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CLINICAL_TRIALS study record update · 2022-11-17Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ)
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CLINICAL_TRIALS study record update · 2021-04-02Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
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CLINICAL_TRIALS study record update · 2021-04-01Hyperkinetic Movements in Patients With Motor Neuron Disease and in Patients With Spinal Muscular Atrophy and Their Response to Treatment With Nusinersen
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CLINICAL_TRIALS study record update · 2018-02-05A Phase II, Mono-center, Placebo-controlled, Double-blind, Crossover Trial to Investigate Effect and Efficacy of Pyridostigmine in Dutch Patients With Spinal Muscular Atrophy Types 2, 3 and 4
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CLINICAL_TRIALS study record update · 2015-03-23Differential Study of Muscle Transcriptome in Patients With Neuromuscular Disease and Control Subjects
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CLINICAL_TRIALS study record update · 2013-02-15Multicentric, Randomized, Double-blind Study Versus Placebo, With Two Parallel Groups Treated to Evaluate the Efficacy and the Tolerance of Riluzole in Children and Young Adults (6 to 20 Years of Age) With SMA. (Type II and Type III)