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Spinal Muscular Atrophy clinical trials in Pennsylvania
8 studies for Spinal Muscular Atrophy are looking for participants at sites in Pennsylvania.
Cities in Pennsylvania
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
RecruitingPhase 4
3 Months – 24 Months · All sexes · Updated 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
RecruitingPhase 2
Up to age 2 · All sexes · Updated 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
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
- Children'S Hospital of Philadelphia, Philadelphia
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)
- Milton S. Hershey Medical Center | Pennsylvania State University_Hershey, Hershey
- Children's Hospital of Philadelphia, Philadelphia
RecruitingPhase 1
Ages 3 and older · All sexes · Updated 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
Recruiting
Ages 3 and older · All sexes · Updated 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
RecruitingObservational study
Ages 18 to 90 · All sexes · Updated May 20, 2026 · NCT05966038Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
- Penn State Hershey, Hershey
- University of Pittsburgh Medical Center, Pittsburgh
RecruitingObservational study
All ages · All sexes · Updated Jan 31, 2025 · NCT04174157From ClinicalTrials.gov, data retrieved Oct 5, 2026. Each study sets its own eligibility; the study team decides who can join.