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Glycogen Storage Disease Type II clinical trials in Philadelphia, PA

4 studies for Glycogen Storage Disease Type II 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.

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A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

RecruitingPhase 1/2
  • Hospital of the University of Pennsylvania, Philadelphia
Ages 18 to 65 · All sexes · Updated Jul 20, 2026 · NCT07282847

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

RecruitingPhase 2
  • University of Pennsylvania, Philadelphia
Ages 18 and older · All sexes · Updated Jun 23, 2026 · NCT07123155

Pompe Disease Registry Protocol

RecruitingObservational study
  • Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089, Philadelphia
  • Children's Hospital of Philadelphia- Site Number : 840034, Philadelphia
All ages · All sexes · Updated Jun 23, 2026 · NCT00231400

A Global Prospective Observational Registry of Patients With Pompe Disease

RecruitingObservational study
  • University of Pennsylvania Perelman Center for Advanced Medicine, Philadelphia
All ages · All sexes · Updated Mar 10, 2026 · NCT06121011

Glycogen Storage Disease Type II 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.