Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2025-05-25Clinical Trial Update: Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab [Phase 3]
Phase 3 extension study assessing long-term safety and efficacy of Apitegromab in spinal muscular atrophy patients who completed prior trial…
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CLINICAL_TRIALS trial_result · 2025-05-02Clinical Trial Update: Two Different Treatment Modalities in Patients With Spinal Muscular Atrophy [NA]
A completed clinical trial (NCT06178653) evaluated two different treatment modalities in patients with spinal muscular atrophy. The study wa…
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CLINICAL_TRIALS trial_result · 2025-04-25Clinical Trial Update: Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This clinical-trial update reports on the long-term follow-up study for spinal muscular atrophy patients treated with AVXS-101 (Zolgensma) a…
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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 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 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 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 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 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…
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CLINICAL_TRIALS trial_result · 2024-09-27Clinical Trial Update: Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural Environment [NA]
A completed clinical trial evaluating the safety and usability of a robotic gait device for children and adolescents with neurological or ne…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1 [Phase 1, Phase 2]
This clinical trial update describes an ongoing Phase 1/2 study evaluating the safety and efficacy of EXG001-307 in patients with spinal mus…
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CLINICAL_TRIALS trial_result · 2024-09-03Clinical Trial Update: Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients [Phase 2, Phase 3]
Phase 2 and Phase 3 clinical trials evaluated short-term and long-term treatment with 4-aminopyridine (4-AP) in ambulatory patients with spi…
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CLINICAL_TRIALS trial_result · 2024-08-28Clinical Trial Update: Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I [EARLY_Phase 1]
A completed early-phase 1 clinical trial evaluated the safety and efficacy of EXG001-307 in patients with Spinal Muscular Atrophy Type I.
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CLINICAL_TRIALS trial_result · 2024-05-31Clinical Trial Update: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) [Phase 4]
Phase 4 OFELIA trial evaluating intravenous AVXS-101 (onasemnogene abeparvovec) in pediatric spinal muscular atrophy has completed with resu…
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CLINICAL_TRIALS trial_result · 2024-04-29Clinical Trial Update: A BHI to Increase Hope Level and Stress Level of Parents With a CMC [NA]
This ClinicalTrials.gov update reports the completion of a study by The Hong Kong Polytechnic University evaluating a behavioral health inte…
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CLINICAL_TRIALS trial_result · 2024-03-05Clinical Trial Update: A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy [Phase 2]
This clinical trial update reports results from a Phase 2 study of risdiplam in infants who are genetically diagnosed and presymptomatic for…
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CLINICAL_TRIALS trial_result · 2024-02-07Clinical Trial Update: Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen [Phase 4]
Phase 4 trial update evaluating risdiplam in patients with spinal muscular atrophy previously treated with nusinersen.
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CLINICAL_TRIALS trial_result · 2024-02-01Clinical Trial Update: WREX Outcome Study
Clinical trial update for the WREX Outcome Study (NCT02218593) completed with posted results.
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CLINICAL_TRIALS trial_result · 2024-01-05Clinical Trial Update: Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) [Phase 3]
Phase 3 trial results for AVXS-101 (OAV101) intravenous gene therapy in pediatric spinal muscular atrophy, with results posted.
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CLINICAL_TRIALS trial_result · 2023-02-16Clinical Trial Update: Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy [Phase 1]
Phase 1 clinical trial evaluating intrathecal administration of onasemnogene abeparvovec-xioi for spinal muscular atrophy was terminated, wi…
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CLINICAL_TRIALS trial_result · 2022-11-17Clinical Trial Update: An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy [Phase 2]
This document reports the completion of a Phase 2 active-treatment trial of SRK-015 in spinal muscular atrophy (types 2 and 3) with results …
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CLINICAL_TRIALS trial_result · 2022-01-11Clinical Trial Update: Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies [Phase 3]
Phase 3 trial of a single-dose gene replacement therapy for spinal muscular atrophy type I with 1-2 SMN2 copies has completed and posted res…
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CLINICAL_TRIALS trial_result · 2022-01-11Clinical Trial Update: Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 [Phase 3]
Phase 3 trial update evaluating intravenous Zolgensma (Onasemnogene abeparvovec-xioi) in presymptomatic spinal muscular atrophy patients wit…