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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
- Children'S Hospital of Philadelphia, Philadelphia
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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
- The Children's Hospital of Philadelphia, Philadelphia
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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
- Children's Hospital Philadelphia - Neurology, Philadelphia
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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
- Children'S Hospital of Philadelphia, Philadelphia
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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)
- Milton S. Hershey Medical Center | Pennsylvania State University_Hershey, Hershey
- Children's Hospital of Philadelphia, Philadelphia
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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
- Penn State Milton S. Hershey Medical Center, Hershey
- Children's Hospital Philadelphia, Philadelphia
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Laj 3 ak plis · Tout sèks · Mizajou Aug 19, 2026 · NCT05866419ALS/MND Natural History Study Data Repository
- Thomas Jefferson University, Philadelphia
- Temple University Lewis Katz School of Medicine, Philadelphia
- University of Pittsburgh, Pittsburgh
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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)
- Penn State Hershey, Hershey
- University of Pittsburgh Medical Center, Pittsburgh
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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.