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Spinal Muscular Atrophy clinical trials in Philadelphia, PA

7 studies for Spinal Muscular Atrophy are looking for participants at sites in and around Philadelphia (within about 30 miles). Open a study to see who can join and how to contact the study team.

Recruiting for a study here? Feature it at the top of this page — $99 for 30 days →

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

RecruitingPhase 2
  • The Children's Hospital of Philadelphia, Philadelphia
Up to age 2 · All sexes · Updated Oct 2, 2026 · NCT07047144

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

RecruitingPhase 4
  • Children'S Hospital of Philadelphia, Philadelphia
3 Months – 24 Months · All sexes · Updated Oct 2, 2026 · NCT05861986

A 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

RecruitingPhase 4
  • Children'S Hospital of Philadelphia, Philadelphia
3 Months – 24 Months · All sexes · Updated Sep 10, 2026 · NCT05861999

A 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)

RecruitingPhase 1
  • Children's Hospital of Philadelphia, Philadelphia
Ages 3 and older · All sexes · Updated Sep 3, 2026 · NCT06555419

Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

Recruiting
  • Children's Hospital Philadelphia, Philadelphia
Ages 3 and older · All sexes · Updated Aug 19, 2026 · NCT05866419

ALS/MND Natural History Study Data Repository

RecruitingObservational study
  • Thomas Jefferson University, Philadelphia
  • Temple University Lewis Katz School of Medicine, Philadelphia
Ages 18 to 90 · All sexes · Updated May 20, 2026 · NCT05966038

About Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) attacks nerve cells in the spinal cord, weakening voluntary muscles. Read about the genetics, types, and what may help.

More about Spinal Muscular Atrophy on MedlinePlus

Spinal Muscular Atrophy trials in other cities

Other studies in Philadelphia

From ClinicalTrials.gov, data retrieved Oct 6, 2026. Each study sets its own eligibility; the study team decides who can join.