Records containing: Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2021-02-18An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2021-02-16An Open-label Study to Assess the Safety and Tolerability of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1
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CLINICAL_TRIALS study record update · 2021-02-16An Open-label Study to Assess the Safety and Tolerability of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in 396443-CS2 or 396443-CS10
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CLINICAL_TRIALS study record update · 2020-11-25Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen: Retro NusiMFM
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CLINICAL_TRIALS study record update · 2020-11-18New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2020-10-19Comparison of the Immunogenicity of Intramuscular Versus Subcutaneous Administration of Trivalent Inactivated Influenza Vaccine in Individuals With Neuromuscular Diseases
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CLINICAL_TRIALS study record update · 2020-10-19A Phase I, 2-Part, Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Doses of Risdiplam and the Effect of Risdiplam on the Pharmacokinetics of Midazolam Following Oral Administration in Healthy Participants
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CLINICAL_TRIALS study record update · 2020-09-02Prospective Evaluation of Infants With Spinal Muscular Atrophy: SPOT SMA
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CLINICAL_TRIALS study record update · 2020-08-31A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2020-08-28A Managed Access Program (MAP) Cohort Treatment Protocol to Provide AVXS-101 to Patients With a Genetic Diagnosis of Spinal Muscular Atrophy (SMA) With 1, 2 or 3 Copies of SMN2
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CLINICAL_TRIALS study record update · 2020-05-14The Burden of Primary Caregivers of Spinal Muscular Atrophy Patients and Their Needs
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CLINICAL_TRIALS study record update · 2020-02-28Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI)
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CLINICAL_TRIALS study record update · 2020-02-21Mechanisms of Cell Death in Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2020-01-27A Phase 2, Randomized, Double-blind, Sham-procedure Controlled Study to Assess the Safety and Tolerability and Explore the Efficacy of ISIS 396443 (BIIB058) Administered Intrathecally in Subjects With Spinal Muscular Atrophy Who Are Not Eligible to Participate in the Clinical Studies ISIS 396443-CS3B or ISIS 396443-CS4
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CLINICAL_TRIALS study record update · 2019-12-12A Pilot Study of the Natural History of Infants With Spinal Muscular Atrophy (SMA) Type 1
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CLINICAL_TRIALS study record update · 2019-12-05SMN Copy Number Distribution in Mali, West Africa
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CLINICAL_TRIALS study record update · 2019-11-25Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2019-09-23A Registered Cohort Study on Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2019-07-26Natural History of Types 2 and 3 Spinal Muscular Atrophy in Taiwan
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CLINICAL_TRIALS study record update · 2019-07-24Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene Aiming at Improving the Genetic Counseling in Spinal Muscular Atrophy Families
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CLINICAL_TRIALS study record update · 2019-05-31Fedt og Sukkerstofskiftet Under Faste Hos Patienter Med Lav Muskelmasse.
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CLINICAL_TRIALS study record update · 2019-02-15Assessment of Feasibility and Statistical Reliability of Functional Outcomes Measurement in Neuromuscular Patients Without Trial Site Visiting by Standard Functional Scales and by Special Autonomic Device in Double-blind, Placebo Controlled Study of Cervical Spinal Cord Transdermal Direct Current Stimulation in Patients With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2019-01-17Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2018-10-30A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy
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CLINICAL_TRIALS study record update · 2018-10-04A Single-Center, Randomized, Investigator/Subject-Blind, Adaptive Single-Ascending-Dose(SAD), Placebo-Controlled, Parallel Study to Investigate the Safety, Tolerability, Pharmacokinetics (Including the Effect of Food and the Effect of Itraconazole on the Pharmacokinetics of a Single Oral Dose of RO7034067), and Pharmacodynamics of RO7034067 Following Oral Administration in Healthy Subjects