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Spinal Muscular Atrophy clinical trials in Florida
8 studies for Spinal Muscular Atrophy are looking for participants at sites in Florida.
Cities in Florida
A 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
RecruitingPhase 2
Ages 5 to 17 · All sexes · Updated 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
RecruitingPhase 4
3 Months – 24 Months · All sexes · Updated 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
RecruitingPhase 1
Ages 3 and older · All sexes · Updated Sep 3, 2026 · NCT06555419A 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
RecruitingPhase 4
3 Months – 24 Months · All sexes · Updated Sep 3, 2026 · NCT05861986Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
- Nemours Children's Hospital, Florida, Orlando
Recruiting
Ages 3 and older · All sexes · Updated Aug 19, 2026 · NCT05866419Clinical Procedures to Support Research in ALS
- University of Miami, Miami
RecruitingObservational study
Ages 18 and older · All sexes · Updated Jun 15, 2026 · NCT03489278ALS/MND Natural History Study Data Repository
- University of Florida, Gainesville
RecruitingObservational study
Ages 18 to 90 · All sexes · Updated 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
RecruitingObservational study
All ages · All sexes · Updated Jan 31, 2025 · NCT04174157From ClinicalTrials.gov, data retrieved Oct 1, 2026. Each study sets its own eligibility; the study team decides who can join.