NCT00485511 |
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A Trial of Hydroxyurea in Spinal Muscular Atrophy
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NCT03837184 |
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Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies
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NCT01671384 |
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Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
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NCT02550691 |
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Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene
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NCT01059240 |
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SMN Copy Number Distribution in Mali West Africa
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NCT05760209 |
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SMN Circular RNAs as Potential New Targets and Biomarkers for SMA
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NCT05544994 |
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The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy
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NCT00155168 |
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Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System
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NCT03381729 |
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Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
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NCT03461289 |
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Single-Dose Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
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NCT05769465 |
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MAP THE SMA a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
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NCT03306277 |
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Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
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NCT03505099 |
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Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy SMA for Patients With Multiple Copies of SMN2
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NCT05335876 |
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Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
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NCT03955679 |
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AveXis Managed Access Program Cohort for Access to AVXS-101
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NCT04317196 |
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Association of Copy Number Variations in the SMN Genes With Acute Kidney Injury After Heart Surgery
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NCT05861999 |
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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
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NCT05861986 |
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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
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NCT02876094 |
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Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy
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