Late-onset Pompe Disease
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) [Phase 2]
Shionogi is sponsoring a Phase 2 study of S-606001 as an add-on to enzyme replacement therapy in adults with late-onset Pompe disease. The t…
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CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: 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) [Phase 1, Phase 2]
A clinical trial update announcing a Phase 1/2 study to evaluate the safety, tolerability, and efficacy of AB-1009 gene therapy for adult pa…
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CLINICAL_TRIALS research · 2026-01-21Clinical Trial Update: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease [Phase 1]
A Phase 1 clinical trial update describes safety, pharmacokinetics, and pharmacodynamics evaluation of DNL952 in adults with late-onset Pomp…
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CLINICAL_TRIALS trial_result · 2026-01-12Clinical 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.
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CLINICAL_TRIALS research · 2025-12-26Clinical Trial Update: Study to Assess the Safety, Tolerability, PK and PD of ABX1100 [EARLY_Phase 1]
A Phase 1 study assessing the safety, tolerability, pharmacokinetics and pharmacodynamics of ABX1100 in healthy volunteers and in individual…
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CLINICAL_TRIALS trial_result · 2025-10-01Clinical 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…
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CLINICAL_TRIALS research · 2025-09-22Clinical Trial Update: Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
A Duke University-sponsored clinical trial update discusses developing a management approach for patients with late-onset Pompe disease.
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CLINICAL_TRIALS trial_result · 2025-07-08Clinical 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.
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CLINICAL_TRIALS trial_result · 2023-01-09Clinical 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.
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CLINICAL_TRIALS trial_result · 2018-06-14Clinical 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 research · 2017-03-20Clinical Trial Update: Search for Serum/Plasma Biomarkers in Pompe's Disease
A clinical trial is investigating serum/plasma biomarkers in Pompe disease, including late-onset Pompe disease, to advance biomarker discove…
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CLINICAL_TRIALS trial_result · 2016-10-31Clinical 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…
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CLINICAL_TRIALS trial_result · 2014-12-02Clinical 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…