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 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 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 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…