Late-onset Pompe Disease

• CLINICAL_TRIALS trial_result · 2026-01-12
  Clinical Trial Update: A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) [Phase 3]

  Phase 3 study of ATB200/AT2221 in adults with late-onset Pompe disease has completed, with long-term safety and efficacy results posted.

• CLINICAL_TRIALS trial_result · 2025-10-01
  Clinical Trial Update: High Dose Inspiratory Muscle Training in LOPD [NA]

  Clinical trial update on high-dose inspiratory muscle training for late-onset Pompe disease, a lysosomal storage disorder, with the study st…

• CLINICAL_TRIALS trial_result · 2025-07-08
  Clinical Trial Update: STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

  The STRIDE study evaluating AT-GAA in patients with late-onset Pompe disease who are on enzyme replacement therapy has been terminated.

• CLINICAL_TRIALS trial_result · 2023-01-09
  Clinical Trial Update: A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

  A clinical trial studying respiratory muscle strength in patients with late-onset Pompe disease was terminated; results have been posted.

• CLINICAL_TRIALS trial_result · 2018-06-14
  Clinical Trial Update: BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) [Phase 3]

  Phase 3 INSPIRE study of BMN 701 in rhGAA-exposed subjects with late-onset Pompe disease was terminated and results were posted in 2018.

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• CLINICAL_TRIALS trial_result · 2016-10-31
  Clinical Trial Update: A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

  This is an update on a prospective, noninterventional observational study of late-onset Pompe disease sponsored by BioMarin; the study evalu…

• CLINICAL_TRIALS trial_result · 2014-12-02
  Clinical Trial Update: Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa [Phase 4]

  Phase 4 exploratory study assessing muscle biopsy changes in patients with late-onset Pompe disease treated with alglucosidase alfa; results…