Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2021-06-15Clinical Trial Update: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants [Phase 2]
Phase 2 trial evaluating risdiplam in Type 2 and 3 Spinal Muscular Atrophy completed with results posted in 2021.
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CLINICAL_TRIALS trial_result · 2021-04-02Clinical Trial Update: Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 [Phase 3]
A Phase 3 trial of a single-dose gene replacement therapy for Spinal Muscular Atrophy Type 1 has completed, with results posted in 2021. The…
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CLINICAL_TRIALS trial_result · 2021-02-21Clinical Trial Update: A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function [Phase 1]
Phase 1 pharmacokinetics and safety study evaluating hepatic impairment on risdiplam after a single oral dose, comparing impaired participan…
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CLINICAL_TRIALS trial_result · 2021-01-08Clinical Trial Update: Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy [Phase 2]
A Phase 2 trial of risdiplam in infants with Spinal muscular atrophy completed, reporting safety, tolerability, PK/PD and efficacy with resu…
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Flu Vaccine Study in Neuromuscular Patients 2011 [Phase 4]
A Phase 4 clinical trial update on a flu vaccine study in pediatric neuromuscular disease patients has completed, with results posted in 202…
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants [Phase 1]
Phase 1 study evaluating drug-drug interaction between risdiplam (multiple dose) and midazolam in healthy participants with spinal muscular …
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CLINICAL_TRIALS trial_result · 2020-08-31Clinical Trial Update: A Study of CK-2127107 in Patients With Spinal Muscular Atrophy [Phase 2]
A Phase 2 clinical trial of CK-2127107 in patients with spinal muscular atrophy has completed with results posted.
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CLINICAL_TRIALS trial_result · 2020-07-16Clinical Trial Update: Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 [Phase 3]
Phase 3 trial results for a gene replacement therapy in spinal muscular atrophy type 1 were completed, with results posted in July 2020. The…
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CLINICAL_TRIALS trial_result · 2020-01-27Clinical Trial Update: A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). [Phase 2]
Phase 2 trial assessing safety and tolerability of nusinersen (ISIS 396443) in participants with spinal muscular atrophy was terminated; res…
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CLINICAL_TRIALS trial_result · 2019-10-02Clinical Trial Update: A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients [Phase 1, Phase 2]
Pilot therapeutic trial evaluating hydroxyurea in Type II and Type III spinal muscular atrophy patients completed.
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CLINICAL_TRIALS trial_result · 2019-10-02Clinical Trial Update: A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients [Phase 1, Phase 2]
Pilot therapeutic trial evaluating hydroxyurea in patients with Type I spinal muscular atrophy (Phase 1/Phase 2) completed with results post…
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CLINICAL_TRIALS trial_result · 2019-08-09Clinical Trial Update: A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) [Phase 2]
Phase 2 study evaluating long-term safety, tolerability, and effectiveness of olesoxime in patients with spinal muscular atrophy has complet…
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CLINICAL_TRIALS trial_result · 2018-10-30Clinical Trial Update: A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) [Phase 2]
Phase 2 clinical trial evaluating nusinersen (ISIS 396443) for efficacy, safety, and pharmacokinetics in infants with spinal muscular atroph…
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CLINICAL_TRIALS trial_result · 2018-05-04Clinical Trial Update: Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
This completed clinical trial studied biomarkers in the immediate postnatal period of development in Spinal Muscular Atrophy; results were p…
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CLINICAL_TRIALS trial_result · 2018-02-22Clinical Trial Update: A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) [Phase 3]
Phase 3 trial evaluating nusinersen (ISIS 396443) for later-onset spinal muscular atrophy has been completed with results posted.
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CLINICAL_TRIALS trial_result · 2018-02-05Clinical Trial Update: SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 [Phase 2]
Phase 2 trial comparing pyridostigmine to placebo in Spinal Muscular Atrophy types 2, 3, and 4 has completed.
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CLINICAL_TRIALS trial_result · 2017-07-28Clinical Trial Update: A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy [Phase 3]
Phase 3 trial of nusinersen (ISIS 396443) in infants with spinal muscular atrophy was terminated; results posted in 2017.
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CLINICAL_TRIALS trial_result · 2017-06-22Clinical Trial Update: Natural History of Spinal Muscular Atrophy Type 1 in Taiwan
This completed clinical trial in Taiwan documented the natural history of spinal muscular atrophy type 1. It was conducted at Kaohsiung Medi…
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CLINICAL_TRIALS trial_result · 2017-03-13Clinical Trial Update: An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy [Phase 1, Phase 2]
Open-label safety, tolerability, and dose-range finding study of nusinersen in spinal muscular atrophy completing phases 1 and 2 with posted…
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CLINICAL_TRIALS trial_result · 2016-12-22Clinical Trial Update: A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) [Phase 1]
A Phase 1 study of RO6885247 in adult and pediatric patients with spinal muscular atrophy has been terminated.
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CLINICAL_TRIALS trial_result · 2016-12-06Clinical Trial Update: Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy [Phase 2]
A Phase 2 clinical trial evaluating valproic acid in ambulant adults with spinal muscular atrophy has been completed.
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CLINICAL_TRIALS trial_result · 2016-11-02Clinical Trial Update: Ability of Muscle Imaging and Motor Function Measure (MFM) to Detect Changes in Disease Progression in Ambulant Spinal Muscular Atrophy Patients Compared to Healthy Volunteers.
A completed clinical trial evaluated whether muscle imaging and the Motor Function Measure can detect changes in disease progression in ambu…
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CLINICAL_TRIALS trial_result · 2016-11-02Clinical Trial Update: Newborn Screening for Spinal Muscular Atrophy
A completed clinical trial evaluating newborn screening for spinal muscular atrophy.
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CLINICAL_TRIALS trial_result · 2016-01-14Clinical Trial Update: Effects of Power Mobility on Young Children With Severe Motor Impairments [Phase 2]
Phase 2 clinical trial update examining the effects of power mobility on young children with severe motor impairments, including cerebral pa…
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CLINICAL_TRIALS trial_result · 2015-10-29Clinical Trial Update: Aerobic Training in Patients With Spinal Muscular Atrophy Type III [NA]
Clinical trial update noting the completion of a study evaluating aerobic training in patients with Spinal muscular atrophy Type III.