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Home › Conditions › Late-onset Pompe disease

Late-onset Pompe disease

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Late-onset Pompe disease

5 trials tracked for Late-onset Pompe disease: 5 in phase 3 or 4 and 2 with published results. The most-cited published study has 24 citations.

5Trials tracked
5Phase 3 & 4
0Recruiting
2With published results
Phase distribution
Phase 4 2 Phase 3 3
  1. Phase 3 A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) Completed · 24 cited
  2. Phase 3 A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease Completed · 8 cited
  3. Phase 4 Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa Completed
  4. Phase 4 Late-Onset Treatment Study Extension Protocol Completed
  5. Phase 3 A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease Completed

Showing the 5 most-cited and recently-updated of 5 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Retrospective cohort shows ERT associated with lower mortality in Chinese late-onset Pompe disease patients
Nutrition & Obesity Medicine Cohort
Retrospective cohort shows ERT associated with lower mortality in Chinese late-onset Pompe disease patients Enzyme therapy saves lives in rare muscle disease patients
This retrospective cohort study included 68 Chinese patients with late-onset Pompe disease followed for a 17-year period.
New data shows enzyme therapy drastically lowers death risk for people with late-onset Pompe disease, especially when started early.
Frontiers Apr 28, 2026