Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2025-07-18Clinical Trial Update: The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. [NA]
This clinical trial update outlines a study evaluating whether assistive gait devices, following a training period, can reduce fall risk in …
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CLINICAL_TRIALS research · 2025-07-17Clinical Trial Update: Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
This is an observational postmarketing surveillance study of nusinersen (Spinraza) for spinal muscular atrophy. It is sponsored by Biogen an…
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CLINICAL_TRIALS research · 2025-07-11Clinical Trial Update: Adult Spinal Muscular Atrophy (SMA) China Registry
Biogen-sponsored adult spinal muscular atrophy registry in China (NCT05618379) is currently active but not recruiting.
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CLINICAL_TRIALS research · 2025-07-09Clinical Trial Update: A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec [Phase 1]
Phase 1 study update evaluating the safety and pharmacokinetics of BIIB115 in healthy adult volunteers and in pediatric spinal muscular atro…
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CLINICAL_TRIALS research · 2025-07-08Clinical Trial Update: A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular Atrophy [NA]
A registry-based randomized controlled trial protocol evaluating an upper-limb exergaming intervention for children and adolescents with spi…
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CLINICAL_TRIALS research · 2025-07-03Clinical Trial Update: Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients [Phase 1, Phase 2]
A clinical trial update indicates a gene therapy drug is being evaluated for safety and efficacy in Spinal Muscular Atrophy Type 1, currentl…
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CLINICAL_TRIALS research · 2025-06-18Clinical Trial Update: Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy [NA]
This clinical trial update reports on the safety and tolerability of low motoneuron stimulation via transcranial magnetic stimulation in spi…
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CLINICAL_TRIALS research · 2025-06-05Clinical Trial Update: Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III [Phase 3]
Phase III clinical trial evaluating GC101 gene therapy via intrathecal injection for type 2 spinal muscular atrophy; currently recruiting.
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CLINICAL_TRIALS research · 2025-05-18Clinical Trial Update: Adult SMA Research and Clinical Hub
Clinical trial update indicating ongoing recruitment for an adult spinal muscular atrophy study in the UK (SMA Research and Clinical Hub).
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CLINICAL_TRIALS research · 2025-05-06Clinical Trial Update: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) [Phase 3]
An ongoing Phase 3 clinical trial is evaluating higher doses of nusinersen (BIIB058) given by injections in SMA patients who previously rece…
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CLINICAL_TRIALS research · 2025-04-29Clinical Trial Update: COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis [NA]
Update on Synchron Medical's COMMAND Early Feasibility Study, an implantable brain-computer interface to help people with paralysis control …
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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 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 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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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 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 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…