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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NCT02122952 |
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Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1
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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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NCT05481164 |
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Newborn Screening for Spinal Muscular Atrophy
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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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NCT04576494 |
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Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults SMA Type 2 or 3 Forms
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NCT04851873 |
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Safety and Efficacy of Intravenous OAV101 AVXS-101 in Pediatric Patients With Spinal Muscular Atrophy SMA
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NCT01476462 |
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Second Malignant Neoplasms After Childhood ALL Therapy
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NCT02941328 |
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SPACE Trial Pyridostigmine vs Placebo in SMA Types 2 3 and 4
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NCT05237544 |
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Newborn Screening for Spinal Muscular Atrophy SMA - a Proof of Principle Study Using Anonymised Blood Spots
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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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NCT05475691 |
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Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America
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NCT02466529 |
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Natural History of Spinal Muscular Atrophy Type 1 in Taiwan
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NCT05614531 |
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Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1
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NCT04292574 |
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UK SMA Patient Registry
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NCT03217578 |
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Neonatal Spinal Muscular Atrophy SMA Screening
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NCT06147414 |
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Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
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NCT06310421 |
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Spinal Muscular Atrophy Neonatal Screening Program
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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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NCT04174157 |
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Registry of Patients With a Diagnosis of Spinal Muscular Atrophy SMA
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NCT06562283 |
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Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
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NCT05073133 |
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Safety and Efficacy of Intravenous OAV101 AVXS-101 in Pediatric Patients With Spinal Muscular Atrophy SMA OFELIA
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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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NCT04404764 |
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Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III Observational Study
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NCT05366465 |
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Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France
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NCT00061607 |
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Measuring Levels of SMN in Blood Samples of SMA Patients
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NCT02123186 |
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Newborn Screening for Spinal Muscular Atrophy
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NCT02855112 |
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Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients
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