Pompe Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2025-07-18Clinical Trial Update: The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. [NA]
This clinical trial update outlines a study evaluating whether assistive gait devices, following a training period, can reduce fall risk in …
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CLINICAL_TRIALS research · 2025-07-03Clinical Trial Update: Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug [Phase 1, Phase 2]
This clinical trial update describes an investigational gene therapy for late-onset Pompe disease being evaluated in Phase 1/2, currently re…
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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 research · 2025-05-13Clinical Trial Update: A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
This clinical trial update describes a prospective study to observe clinical outcomes of alglucosidase alfa treatment in infants ≤6 months w…
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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 research · 2025-02-18Clinical Trial Update: Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases [NA]
A recruiting clinical trial using transcriptomic analysis to reduce misdiagnosis in patients with rare genetic muscle diseases, including Du…
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EMA approval · 2025-02-14EMA Authorised: Opfolda (miglustat) — Amicus Therapeutics Europe Limited
The EMA authorised Opfolda (miglustat) for the treatment of Pompe disease (Glycogen Storage Disease Type II), with Amicus Therapeutics Europ…
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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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FDA approval · 2024-12-30FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Lumizyme (alglucosidase alfa) by Genzyme Corporation.
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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 research · 2024-08-28Clinical Trial Update: Treatment Frequency Reduction in Pompe Disease [Phase 4]
Phase 4 clinical trial update on reducing treatment frequency for Pompe disease (late-onset); recruitment has not yet started.
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EMA approval · 2024-08-16EMA Authorised: Myozyme (alglucosidase alfa) — Sanofi B.V.
EMA authorised marketing of Myozyme (alglucosidase alfa) by Sanofi B.V. for Glycogen storage disease type II (Pompe disease).
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FDA approval · 2024-07-25FDA Approved: POMBILITI ATGA — Supplemental Application (AMICUS THERAPEUTICS US, LLC)
The FDA approved a supplemental application for POMBILITI ATGA (cipaglucosidase alfa-ATGA) from Amicus Therapeutics US, LLC. This action upd…
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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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FDA approval · 2024-03-22FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation.
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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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FDA approval · 2023-09-28FDA Approved: POMBILITI ATGA — Original Application (AMICUS THERAPEUTICS US, LLC)
FDA approves POMBILITI ATGA (cipaglucosidase alfa-ATGA) as an original biologics license application for Pompe disease.
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FDA approval · 2023-09-15FDA Approved: NEXVIAZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for NEXVIAZYME (avalglucosidase alfa-ngpt) submitted by Genzyme Corporation. This action updates the…
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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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FDA approval · 2023-05-24FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
The FDA approved a supplemental application for Lumizyme (alglucosidase alfa) submitted by Genzyme Corporation.
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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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FDA approval · 2022-05-12FDA Approved: LUMIZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for Lumizyme (alglucosidase alfa) from Genzyme Corporation. The action pertains to Pompe disease.
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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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FDA approval · 2021-08-06FDA Approved: NEXVIAZYME — Original Application (Genzyme Corporation)
FDA approved Nexviazyme (avalglucosidase alfa-ngpt) from Genzyme Corporation under the original BLA. It is indicated for Pompe disease.