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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
- The University of Texas Southwestern Medical Center at Dallas, Dallas
- Cook Children's Jane and John Justin Neurosciences Center, Fort Worth
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3 Months – 24 Months · Tout sèks · Mizajou Oct 2, 2026 · NCT05861986A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
- University of Texas Southwestern Medical Center, Dallas
- Neurology Rare Disease Center, Flower Mound
- Cook Children's Medical Center, Fort Worth
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Jiska laj 2 · Tout sèks · Mizajou Oct 2, 2026 · NCT07047144A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
- Neurology Rare Disease Center, Flower Mound
- Texas Children's Hospital, Houston
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Laj 5 a 17 · Tout sèks · Mizajou Sep 14, 2026 · NCT07287982A 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
- University of Texas Southwestern Medical Center, Dallas
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3 Months – 24 Months · Tout sèks · Mizajou Sep 10, 2026 · NCT05861999A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
- Texas Childrens Hospital Houston, Houston
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Laj 3 ak plis · Tout sèks · Mizajou Sep 3, 2026 · NCT06555419Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
- Texas Children's Hospital, Houston
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Laj 3 ak plis · Tout sèks · Mizajou Aug 19, 2026 · NCT05866419A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
- Neurology Rare Disease Center, Flower Mound
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0 Days – 42 Days · Tout sèks · Mizajou Aug 18, 2026 · NCT07221669A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
- Neurology Rare Disease Center, Flower Mound
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0 Days – 7 Months · Tout sèks · Mizajou Aug 11, 2026 · NCT07444450Clinical Procedures to Support Research in ALS
- University of Texas San Antonio Health Science Center, San Antonio
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Laj 18 ak plis · Tout sèks · Mizajou Jun 15, 2026 · NCT03489278Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
- Cook Children's, Fort Worth
- Texas Children's Hospital, Houston
- CHRISTUS Health, San Antonio
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Tout laj · Tout sèks · Mizajou Jan 31, 2025 · NCT04174157Sous ClinicalTrials.gov, done yo ranmase Oct 5, 2026. Chak etid gen kritè li pou li menm; ekip etid la deside ki moun ki ka patisipe.