Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2025-09-15Clinical Trial Update: Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies [NA]
An ongoing study assessing motor function in infants with spinal muscular atrophy who are being treated with innovative therapies; the trial…
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CLINICAL_TRIALS research · 2025-09-15Clinical Trial Update: Pediatric Powered Wheelchair Standing Devices: An Exploratory Study [NA]
Exploratory study of pediatric powered wheelchair standing devices. The trial is currently active but not recruiting and focuses on children…
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CLINICAL_TRIALS trial_result · 2025-09-11Clinical Trial Update: Palliative Care in Spinal Muscular Atrophy (SMA) 1 [NA]
This clinical trial update notes the completion of a study on palliative care in Spinal Muscular Atrophy type 1.
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CLINICAL_TRIALS research · 2025-09-03Clinical Trial Update: Pediatric Spinal Muscular Atrophy (SMA) China Registry
Biogen-sponsored pediatric spinal muscular atrophy registry in China is currently active and not recruiting.
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CLINICAL_TRIALS research · 2025-08-29Clinical Trial Update: Robot-assisted Training [NA]
A clinical trial conducted by Peking University Third Hospital is evaluating robot-assisted training for spinal muscular atrophy and is curr…
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EMA approval · 2025-08-28EMA Authorised: Zolgensma (onasemnogene abeparvovec) — Novartis Europharm Limited
The EMA authorised Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy under conditional, orphan designation, with Novartis Eur…
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CLINICAL_TRIALS research · 2025-08-26Clinical Trial Update: Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb [NA]
A clinical trial evaluating spinal cord stimulation to improve upper-limb motor function in spinal muscular atrophy is currently active but …
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CLINICAL_TRIALS research · 2025-07-22Clinical Trial Update: An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy [Phase 1, Phase 2]
Open-label Phase 1/2 trial evaluating Vesemnogene Lantuparvovec for spinal muscular atrophy; currently recruiting.
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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 trial_result · 2025-06-05Clinical Trial Update: Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy [Phase 3]
Phase 3 clinical trial results for nusinersen (BIIB058) in spinal muscular atrophy have been completed. The results were posted on 2025-06-0…
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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 trial_result · 2025-06-03Clinical Trial Update: Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam [Phase 3]
Phase IIIb, open-label, multicenter study evaluating the safety, tolerability, and efficacy of intrathecal OAV101 in spinal muscular atrophy…
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CLINICAL_TRIALS trial_result · 2025-05-28Clinical Trial Update: Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV [NA]
An exploratory study evaluating respiratory bacterial infections, superinfections, and colonizations in spinal muscular atrophy patients usi…
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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 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 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 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 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…