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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
- University of Florida Pediatrics, Gainesville
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3 Months – 24 Months · Tout sèks · Mizajou Oct 2, 2026 · NCT05861986A 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
- Rare Disease Research FL LLC, Kissimmee
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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 Florida Pediatrics, Gainesville
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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)
- Nemours Children's Hospital, Orlando, Orlando
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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
- Nemours Children's Hospital, Florida, Orlando
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Laj 3 ak plis · Tout sèks · Mizajou Aug 19, 2026 · NCT05866419Clinical Procedures to Support Research in ALS
- University of Miami, Miami
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Laj 18 ak plis · Tout sèks · Mizajou Jun 15, 2026 · NCT03489278ALS/MND Natural History Study Data Repository
- University of Florida, Gainesville
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Laj 18 a 90 · Tout sèks · Mizajou May 20, 2026 · NCT05966038Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
- Golisano Children's Hospital of Southwest Florida, Fort Myers
- Jackson South Medical Center, Palmetto Bay
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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.