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Oficialus tyrimo tekstas (anglų kalba)
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
Renkami dalyviai4 fazė
3 Months – 24 Months · Visoms lytims · Atnaujinta 2026-10-02 · 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
Renkami dalyviai2 fazė
Amžius nuo 5 iki 17 · Visoms lytims · Atnaujinta 2026-09-14 · 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
Renkami dalyviai4 fazė
3 Months – 24 Months · Visoms lytims · Atnaujinta 2026-09-10 · 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
Renkami dalyviai1 fazė
Amžius 3 ir vyresni · Visoms lytims · Atnaujinta 2026-09-03 · NCT06555419Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
- Nemours Children's Hospital, Florida, Orlando
Renkami dalyviai
Amžius 3 ir vyresni · Visoms lytims · Atnaujinta 2026-08-19 · NCT05866419Clinical Procedures to Support Research in ALS
- University of Miami, Miami
Renkami dalyviaiStebimasis tyrimas
Amžius 18 ir vyresni · Visoms lytims · Atnaujinta 2026-06-15 · NCT03489278ALS/MND Natural History Study Data Repository
- University of Florida, Gainesville
Renkami dalyviaiStebimasis tyrimas
Amžius nuo 18 iki 90 · Visoms lytims · Atnaujinta 2026-05-20 · 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
Renkami dalyviaiStebimasis tyrimas
Visi amžiai · Visoms lytims · Atnaujinta 2025-01-31 · NCT04174157Iš ClinicalTrials.gov, duomenys gauti 2026-10-05. Kiekvienas tyrimas nustato savo tinkamumo kriterijus; tyrimo komanda sprendžia, kas gali dalyvauti.