Pompe Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease [Phase 1]
Phase 1 clinical trial ACTUS-101 (AAV2/8-LSPhGAA) for late-onset Pompe disease has completed.
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CLINICAL_TRIALS trial_result · 2026-03-23Clinical Trial Update: China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD [Phase 4]
Phase 4 post-approval commitment study of avalglucosidase alfa for Pompe disease, currently recruiting in China in participants with infanti…
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CLINICAL_TRIALS trial_result · 2026-01-16Clinical Trial Update: Avalglucosidase Alfa Pregnancy Study
The Avalglucosidase alfa pregnancy study (NCT05734521) sponsored by Sanofi is currently recruiting to evaluate its use in Pompe disease duri…
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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 trial_result · 2025-10-23Clinical Trial Update: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 [Phase 1, Phase 2]
First-in-human study evaluating safety, tolerability, and pharmacokinetics of intravenous ATB200 alone and when co-administered with oral AT…
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CLINICAL_TRIALS trial_result · 2025-07-03Clinical Trial Update: Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/2 evaluation of a gene therapy for infantile-onset Pompe disease, with the study currently ac…
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CLINICAL_TRIALS trial_result · 2025-03-12Clinical Trial Update: MSOT in Pompe Disease [NA]
This clinical trial update reports on a study evaluating multispectral optoacoustic tomography (MSOT) in Pompe disease, with the study statu…
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CLINICAL_TRIALS trial_result · 2025-02-10Clinical Trial Update: Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen [Phase 4]
Phase 4 study update evaluating the efficacy and safety of alglucosidase alfa in Chinese patients with late-onset Pompe disease; the trial i…
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CLINICAL_TRIALS trial_result · 2024-11-27Clinical Trial Update: A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) [Phase 1, Phase 2]
Update on the RESOLUTE gene transfer study for late-onset Pompe disease, currently active but not recruiting.
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CLINICAL_TRIALS trial_result · 2024-06-24Clinical Trial Update: Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Completed clinical trial NCT02354664 evaluating dynamic respiratory muscle function in late-onset Pompe disease; results were posted on 2024…
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CLINICAL_TRIALS trial_result · 2023-11-14Clinical Trial Update: Nutritional Therapy in Late-onset Pompe Disease [Phase 2]
Phase 2 trial evaluating nutritional therapy for late-onset Pompe disease with muscle loss; current status is unknown according to ClinicalT…
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CLINICAL_TRIALS trial_result · 2023-09-11Clinical Trial Update: A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease [Phase 3]
Phase 3 study comparing ATB200/AT2221 with alglucosidase alfa/placebo in adults with late-onset Pompe disease has completed, with results po…
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CLINICAL_TRIALS trial_result · 2023-06-05Clinical Trial Update: Response to Diaphragmatic Pacing in Subjects With Pompe Disease
A completed clinical trial investigating diaphragmatic pacing in Pompe disease subjects; results posted in 2023.
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CLINICAL_TRIALS trial_result · 2022-08-26Clinical Trial Update: Growth and Development Study of Alglucosidase Alfa [Phase 4]
A Phase 4 clinical trial update for alglucosidase alfa investigating growth and development in Pompe disease, with results posted after comp…
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CLINICAL_TRIALS trial_result · 2021-11-22Clinical Trial Update: A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa [Phase 2]
Phase 2 pediatric study assessing safety and efficacy of avalglucosidase alfa given every other week in infantile-onset Pompe disease patien…
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CLINICAL_TRIALS trial_result · 2021-06-11Clinical Trial Update: Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease [Phase 4]
Phase 4 pharmacokinetic study of alglucosidase alfa in Pompe disease patients has completed and results were posted in 2021.
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CLINICAL_TRIALS trial_result · 2021-04-08Clinical Trial Update: Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease [Phase 3]
Phase 3 trial comparing avalglucosidase alfa to alglucosidase alfa in late-onset Pompe disease patients who have not been treated before, wi…
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CLINICAL_TRIALS trial_result · 2020-10-12Clinical Trial Update: Immune Tolerance Induction Study [Phase 4]
Phase 4 trial update for Immune Tolerance Induction in Pompe disease was terminated with results posted in 2020.
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CLINICAL_TRIALS trial_result · 2018-08-17Clinical Trial Update: Drug-drug Interaction Study [Phase 2]
Amicus Therapeutics completed a Phase 2 drug–drug interaction study in Pompe disease, and the results have been posted.
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CLINICAL_TRIALS trial_result · 2018-08-17Clinical Trial Update: Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease [Phase 2]
A Phase 2 safety study of AT2220 (duvoglustat) for Pompe disease was terminated, with results posted in 2018.
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CLINICAL_TRIALS trial_result · 2018-06-11Clinical Trial Update: Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease [Phase 1, Phase 2]
BMN701 was studied in late-onset Pompe disease in a Phase 1/2 trial focused on safety, tolerability, pharmacokinetics and pharmacodynamics. …
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CLINICAL_TRIALS trial_result · 2018-05-22Clinical Trial Update: Extension Study for Patients Who Have Participated in a BMN 701 Study [Phase 2]
Extension study of BMN 701 in Pompe disease participants from a prior BMN 701 trial (Phase 2) has been terminated; results were posted in 20…
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CLINICAL_TRIALS trial_result · 2018-05-15Clinical Trial Update: A Pilot Study of Pyridostigmine in Pompe Disease [EARLY_Phase 1]
An early-phase 1 pilot study evaluating pyridostigmine for Pompe disease has been terminated.
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CLINICAL_TRIALS trial_result · 2018-02-06Clinical Trial Update: Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease [Phase 1, Phase 2]
Phase 1/2 trial update evaluating safety and efficacy of albuterol in individuals with Pompe disease has completed with results posted.
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CLINICAL_TRIALS trial_result · 2017-12-08Clinical Trial Update: Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
This completed observational study evaluated subjects with Pompe disease undergoing immune modulation therapies.