Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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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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FDA approval · 2017-11-21FDA Approved: SPINRAZA — Supplemental Application (Biogen Inc.)
FDA approved a supplemental application for Spinraza (nusinersen) filed by Biogen, updating the drug's regulatory status.
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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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EMA approval · 2017-04-21First medicine for spinal muscular atrophy
EMA announces the first medicine approved for spinal muscular atrophy, marking a milestone in treatment for this disease in Europe.
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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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FDA approval · 2016-12-23FDA Approved: SPINRAZA — Original Application (Biogen Inc.)
FDA approved Spinraza (nusinersen) from Biogen in its original application for spinal muscular atrophy.
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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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EMA guidance · 2016-11-07Workshop: working towards new therapies for spinal muscular atrophy
EMA reports on a workshop focused on developing new therapies for spinal muscular atrophy, discussing current challenges and future directio…
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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 research · 2016-11-02Clinical Trial Update: A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
A completed study collected blood samples from patients with spinal muscular atrophy to analyze potential biomarkers.
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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 research · 2016-08-12Clinical Trial Update: Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA) [NA]
This entry notes a completed clinical trial evaluating exercise in patients with spinal muscular atrophy. The study was sponsored by Columbi…
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CLINICAL_TRIALS research · 2016-08-04Clinical Trial Update: Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients [Phase 1, Phase 2]
A Phase 1/2 clinical trial at Tehran University of Medical Sciences is evaluating allogeneic adipose-derived stem cells in infants with spin…
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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 research · 2016-01-01Clinical Trial Update: Valproate and Levocarnitine in Children With Spinal Muscular Atrophy [Phase 3]
Phase 3 clinical trial update evaluating valproate and levocarnitine in children with spinal muscular atrophy.
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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.
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CLINICAL_TRIALS trial_result · 2015-07-03Clinical Trial Update: Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy [Phase 1, Phase 2]
This update reports the completion and posted results of a Phase 1/2 clinical trial evaluating sodium phenylbutyrate in pre-symptomatic infa…
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CLINICAL_TRIALS trial_result · 2015-06-15Clinical Trial Update: CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I [Phase 1, Phase 2]
This clinical trial update reports on a Phase 1/2 study evaluating valproic acid and carnitine in infants with spinal muscular atrophy, with…
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CLINICAL_TRIALS trial_result · 2015-03-23Clinical Trial Update: Differential Study of Muscle Transcriptome [NA]
This update notes that a clinical trial examining differential muscle transcriptome in amyotrophic lateral sclerosis, spinal muscular atroph…
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CLINICAL_TRIALS other · 2014-08-28Clinical Trial Update: Reciprocating Gait Orthoses for Paraplegia Patients [NA]
This entry provides an administrative update on a clinical trial evaluating reciprocating gait orthoses for paraplegia, including patients w…
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CLINICAL_TRIALS research · 2014-04-28Clinical Trial Update: National Registry for Egyptian Pediatric Neuromuscular Diseases
This document provides an update on Egypt's National Registry for Pediatric Neuromuscular Diseases, cataloging Spinal Muscular Atrophy, Musc…