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Examining Sex-based Differences in Metabolic and Mechanistic Responses to Disuse Induced Muscle Atrophy NA The purpose of the proposed research is to define whether there are differe...
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Currie Gynasium 309 - McGill University
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A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I PHASE2 The purpose of the study is to evaluate the long-term safety and tolerabili...
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St. Louis Children's Hospital - Neurology
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Efficacy and Safety of TAK-583 in Subjects With Diabetic Peripheral Neuropathy PHASE2 The purpose of this study is to determine the safety and effectiveness of T...
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Multiple Locations
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Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 PHASE3 To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec...
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David Geffen School of Medicine at UCLA
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Infants With Spinal Muscular Atrophy Type I N/A Background:
\- Spinal muscular atrophy type 1 (SMA 1) causes severe muscle...
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National Institute of Neurological Disorders and Stroke (NINDS), 9000 Rockville
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AMG 745 in Subjects With Age-associated Muscle Loss PHASE2 Randomized, Double-blind, Placebo-controlled Dose Ranging Study to Evaluate...
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Multiple Locations
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Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy N/A The purpose of this study is to investigate the measurement properties of t...
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Multiple Locations
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A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND) PHASE4 In this study, researchers will learn more about the use of nusinersen (BII...
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Arkansas Children's Hospital Research Institute
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Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI) NA This study aims to refine the capability of Multispectral Optoacoustic Tomo...
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University Hospital Erlangen
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French Register of Patients With Spinal Muscular Atrophy N/A The primary objectives of the study are to obtain clinically meaningful dat...
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Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, (French neuromuscular reference network (FILNEMUS), Hôpital Raymond Poincaré,
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