Records containing: Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2018-02-22A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2018-02-05A Phase II, Mono-center, Placebo-controlled, Double-blind, Crossover Trial to Investigate Effect and Efficacy of Pyridostigmine in Dutch Patients With Spinal Muscular Atrophy Types 2, 3 and 4
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CLINICAL_TRIALS study record update · 2017-10-06SMN Levels in Peripheral Blood Samples of SMA Patients and the Effects of Pharmacological Compounds In Vitro
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CLINICAL_TRIALS study record update · 2017-07-28A Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2017-03-13An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2016-12-06Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study
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CLINICAL_TRIALS study record update · 2016-11-02Newborn Screening for Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2016-08-12Randomized, Controlled Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
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CLINICAL_TRIALS study record update · 2016-01-14Effects of Power Mobility on the Development and Function of Young Children With Severe Motor Impairments
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CLINICAL_TRIALS study record update · 2016-01-01Randomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 Years
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CLINICAL_TRIALS study record update · 2015-10-29Aerobic Training Improves Oxidative Capacity, But Not Function in Spinal Muscular Atrophy III
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CLINICAL_TRIALS study record update · 2015-07-03Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2014-08-28NSC Assistive Technology Research: Reciprocating Gait Orthoses for Paraplegia Patients
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CLINICAL_TRIALS study record update · 2014-04-28National Registry for Egyptian Pediatric Neuromuscular Diseases
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CLINICAL_TRIALS study record update · 2014-03-05Clinical Assessment of Spinal Muscular Atrophy Type II and III
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CLINICAL_TRIALS study record update · 2013-11-13Optimum Insufflation Capacity and Peak Cough Flow Augmentation in Patients With Neuromuscular Disease
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CLINICAL_TRIALS study record update · 2012-10-24A Pilot Study of Biomarkers for Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2012-01-31Spinal Muscular Atrophy and Cardiac Autonomic Function
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CLINICAL_TRIALS study record update · 2011-05-03Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial)
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CLINICAL_TRIALS study record update · 2010-02-15A Randomized, Double-Blind, Placebo-Controlled Trial of Hydroxyurea in Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2009-12-16Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy Type II and III Treated With Valproic Acid