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32 studies found for:    Open Studies | "Muscular Atrophy, Spinal"

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1 Not yet recruiting A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (Firefish)
Condition: Muscular Atrophy, Spinal
Intervention: Drug: RO7034067
2 Not yet recruiting A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish)
Condition: Muscular Atrophy, Spinal
Interventions: Drug: Placebo;   Drug: RO7034067
3 Recruiting Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3
Condition: Spinal Muscular Atrophy Type 3
Intervention: Drug: Pyridostigmine Bromide
4 Recruiting Pilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)
Condition: Infantile Spinal Muscular Atrophy of Type 2 or 3
Intervention: Other: Physical exercise in a swimming pool during 6 months
5 Recruiting International SMA Patient Registry
Condition: Muscular Atrophy, Spinal
Intervention:
6 Recruiting Prospective Evaluation of Infants With Spinal Muscular Atrophy:
Condition: Spinal Muscular Atrophy
Intervention:
7 Recruiting Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproate, Levocarnitine;   Drug: Placebo
8 Recruiting A Study of CK-2127107 in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Interventions: Drug: CK-2127107 150 mg;   Drug: Placebo;   Drug: CK-2127107 450 mg
9 Not yet recruiting Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy (SMA)
Intervention: Drug: celecoxib
10 Recruiting A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Nusinersen
11 Not yet recruiting Oxidative Capacity and Exercise Tolerance in Ambulatory SMA
Conditions: Spinal Muscular Atrophy Type 3;   Mitochondrial Myopathy
Intervention:
12 Unknown † Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)
Condition: Spinal Muscular Atrophy
Intervention:
13 Recruiting Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
Conditions: Spinal Muscular Atrophy;   Charcot-Marie-Tooth Disease;   Muscular Dystrophy;   Spinal Muscular Atrophy With Respiratory Distress 1;   Amyotrophic Lateral Sclerosis;   Motor Neuron Disease;   Neuromuscular Disease;   Peroneal Muscular Atrophy;   Fragile X Syndrome
Intervention: Other: sample collection
14 Recruiting A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
Intervention: Drug: Olesoxime
15 Recruiting Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene
Condition: Spinal Muscular Atrophy
Intervention: Procedure: blood sampling
16 Recruiting Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions
Conditions: Osteopenia;   Spinal Muscular Atrophy;   Cerebral Palsy;   Muscular Dystrophy;   Spina Bifida;   Rett Syndrome
Intervention: Other: Assisted Standing Treatment Program
17 Recruiting National Registry for Egyptian Pediatric Neuromuscular Diseases
Conditions: Spinal Muscular Atrophy;   Muscular Dystrophy;   Muscle Diseases;   Myasthenic Syndromes;   Polyneuropathies
Intervention:
18 Unknown † Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System
Condition: Spinal Muscular Atrophy
Intervention:
19 Unknown † Establishing Novel Detection Techniques for Various Genetic-Related Diseases by Applying DHPLC Platform.
Conditions: Spinal Muscular Atrophy;   Neonatal Hyperbilirubinemia;   Colon Cancer
Intervention:
20 Not yet recruiting A Patient Centric Motor Neuron Disease Activities of Daily Living Scale
Conditions: Amyotrophic Lateral Sclerosis;   Progressive Muscular Atrophy;   Primary Lateral Sclerosis;   Hereditary Spastic Paraplegia
Intervention: Other: Web-based Survey
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† Indicates status has not been verified in more than two years