Spinal Muscular Atrophy
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CLINICAL_TRIALS trial_result · 2025-04-25Clinical Trial Update: Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi [Phase 3]
Long-term follow-up study in patients treated with onasemnogene abeparvovec-xioi in a Phase 3 trial; the study is currently active but not r…
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CLINICAL_TRIALS trial_result · 2025-04-09Clinical Trial Update: An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) [Phase 1, Phase 2]
An open-label Phase 1/2 study of branaplam (LMI070) in patients with type 1 spinal muscular atrophy has completed, with results posted.
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CLINICAL_TRIALS research · 2025-04-09Clinical Trial Update: NuroSleeve Powered Brace & Stimulation System to Restore Arm Function [NA]
Clinical trial update for the NuroSleeve Powered Brace & Stimulation System aimed at restoring arm function in people with various neurologi…
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CLINICAL_TRIALS research · 2025-04-06Clinical Trial Update: French Register of Patients With Spinal Muscular Atrophy
The document indicates that the French Spinal Muscular Atrophy patient registry (NCT04177134) is currently recruiting, collecting data on SM…
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CLINICAL_TRIALS research · 2025-04-04Clinical Trial Update: Early Check: Expanded Screening in Newborns
An update on an ongoing clinical trial (NCT03655223) evaluating expanded newborn screening under the Early Check program, with enrollment by…
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CLINICAL_TRIALS trial_result · 2025-04-02Clinical Trial Update: Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular Atrophy [NA]
This clinical trial update reports the completion of a study evaluating virtual reality as a method to mitigate anxiety during intrathecal a…
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CLINICAL_TRIALS research · 2025-03-21Clinical Trial Update: Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy [EARLY_Phase 1]
Clinical trial update on EXG001-307 for Spinal Muscular Atrophy, an early-phase 1 study evaluating safety and efficacy.
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CLINICAL_TRIALS other · 2025-03-14Clinical Trial Update: EAP of Apitegromab for Patients With Spinal Muscular Atrophy
This document provides an update on the expanded access program for Apitegromab in patients with spinal muscular atrophy.
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CLINICAL_TRIALS trial_result · 2025-03-07Clinical Trial Update: Impact of Motor and Oral Motor Function on Quality of Life in Children With SMA
The trial studied how motor and oral motor function relate to quality of life in children with Spinal muscular atrophy and has completed.
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CLINICAL_TRIALS research · 2025-03-07Clinical Trial Update: GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy [NA]
This trial update states that GCB-001 for spinal muscular atrophy type 2 is currently enrolling by invitation.
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CLINICAL_TRIALS research · 2025-03-07Clinical Trial Update: Quality of Life and Participation of the Adult with Spinal Muscular Atrophy in France
A completed clinical trial in France evaluating quality of life and participation of adults with spinal muscular atrophy. The study was spon…
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CLINICAL_TRIALS research · 2025-03-05Clinical Trial Update: A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
A long-term follow-up study is evaluating intravenous EXG001-307 in patients with Type 1 spinal muscular atrophy; the study is currently enr…
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CLINICAL_TRIALS research · 2025-02-12Clinical Trial Update: Holter of Movement in Patients with SMA Undergoing Treatment. [NA]
This clinical trial update indicates an active, not-recruiting study evaluating movement monitoring using Holter methods in patients with sp…
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FDA approval · 2025-02-11FDA Approved: EVRYSDI — Original Application (Genentech, Inc.)
FDA approved Evrysdi (risdiplam) in an Original Application, establishing it as an oral treatment option for spinal muscular atrophy. The ap…
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FDA approval · 2025-02-11FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
FDA approved a supplemental application for EVRYSDI (risdiplam) from Genentech, Inc.
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CLINICAL_TRIALS research · 2025-02-03Clinical Trial Update: Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA). [NA]
A clinical trial update evaluating an optimized rehabilitation program for patients with spinal muscular atrophy, focusing on acceptability,…
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CLINICAL_TRIALS research · 2025-01-31Clinical Trial Update: Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
A registry study sponsored by Novartis to collect information on patients diagnosed with spinal muscular atrophy; the study is currently rec…
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CLINICAL_TRIALS research · 2025-01-29Clinical Trial Update: Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.
A registry study evaluating assistive devices, medicines, and healthcare measures for amyotrophic lateral sclerosis, spinal muscular atrophy…
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CLINICAL_TRIALS trial_result · 2025-01-15Clinical Trial Update: A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
A long-term safety study in Brazilian patients with spinal muscular atrophy treated with Zolgensma is currently recruiting.
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CLINICAL_TRIALS research · 2025-01-03Clinical Trial Update: Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases
A pediatric neuromuscular disease trial is recruiting to evaluate respiratory function, thoracoabdominal movements, and exercise capacity.
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CLINICAL_TRIALS research · 2024-12-19Clinical Trial Update: Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
A clinical trial update reports completion of a genetics study on pediatric-onset motor neuron and neuromuscular diseases, including spinal …
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CLINICAL_TRIALS trial_result · 2024-12-19Clinical Trial Update: Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy [NA]
A clinical trial update indicating a study to evaluate the reproducibility of a fatigability test in patients with spinal muscular atrophy; …
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CLINICAL_TRIALS trial_result · 2024-12-04Clinical Trial Update: Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) [NA]
An update on the functional effects of nusinersen in adults with 5q-spinal muscular atrophy (SMA Type 2 or 3). The study has completed.
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CLINICAL_TRIALS research · 2024-11-20Clinical Trial Update: Spinraza in Adult Spinal Muscular Atrophy
Clinical trial update showing Spinraza (nusinersen) being studied in adults with Spinal Muscular Atrophy, including SMA Type II and SMA Type…
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CLINICAL_TRIALS trial_result · 2024-10-22Clinical Trial Update: A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies [Phase 3]
Phase 3 trial update for spinal muscular atrophy patients who previously participated in nusinersen investigational studies; results have be…