Pompe Disease
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Sign up freeTop drugs studied for this 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 · 2026-04-16Clinical Trial Update: Pompe Pregnancy Sub-Registry
Genzyme sponsors a Pompe disease pregnancy registry that is currently recruiting.
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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 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 · 2026-04-08Clinical Trial Update: A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease [NA]
This clinical trial update describes a study assessing antibody levels and blood biomarkers in people with late-onset Pompe disease, sponsor…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa [Phase 3]
Sanofi is running a Phase 3 clinical trial to evaluate avalglucosidase alfa for treatment-naïve infants with Pompe disease; the trial is cur…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Clinical trial update noting ongoing recruitment for a study assessing CRIM status and longitudinal outcomes in individuals with Pompe disea…
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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 research · 2026-03-17Clinical Trial Update: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) [Phase 1]
Phase 1 prenatal enzyme replacement therapy trial PEARL is currently recruiting for multiple lysosomal storage disorders including MPS I, MP…
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease [NA]
A clinical trial is evaluating the effects of whole-body electrical muscle stimulation on adults with various neuromuscular diseases and is …
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CLINICAL_TRIALS research · 2026-03-10Clinical Trial Update: A Global Prospective Observational Registry of Patients With Pompe Disease
Amicus Therapeutics is conducting a global prospective observational registry of patients with Pompe disease, currently recruiting.
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CLINICAL_TRIALS research · 2026-03-04Clinical Trial Update: Cognitive and Neurological Pathologies in Pompe Disease
A clinical trial update indicates a study examining cognitive and neurological issues in Pompe disease is currently recruiting.
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CLINICAL_TRIALS research · 2026-02-02Clinical Trial Update: Gene Transfer Study in Patients With Late Onset Pompe Disease [Phase 1, Phase 2]
This clinical trial update describes an ongoing phase 1/2 gene transfer study for late-onset Pompe disease sponsored by Astellas Gene Therap…
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EMA approval · 2026-01-23EMA Authorised: Nexviadyme (avalglucosidase alfa) — Sanofi B.V.
EMA authorised Nexviadyme (avalglucosidase alfa) from Sanofi B.V. for Pompe disease (Glycogen Storage Disease Type II) with decision dated 0…
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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 · 2026-01-16Clinical 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 research · 2025-10-31Clinical Trial Update: Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) [Phase 4]
Post-trial access update for avalglucosidase alfa in participants with Pompe disease from a Phase 4 trial; the study is active but not recru…
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CLINICAL_TRIALS research · 2025-10-27Clinical Trial Update: ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD [Phase 3]
This is a Phase 3 pediatric trial (ZIP Study-OL) evaluating safety, pharmacokinetics/pharmacodynamics, efficacy, and immunogenicity of ATB20…
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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 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 · 2025-09-15Clinical Trial Update: Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
Expanded Access update for Amicus Therapeutics' ATB200/AT2221 in Pompe disease. The document notes that access is AVAILABLE under the progra…
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CLINICAL_TRIALS research · 2025-09-10Clinical Trial Update: Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Development and validation of the FBIndex, a tool to assess falls risk in patients with various neuromuscular disorders.
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CLINICAL_TRIALS research · 2025-08-26Clinical Trial Update: Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.
This clinical trial update describes a non-invasive magnetic resonance exploratory pilot study investigating metabolic muscular biomarkers i…
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CLINICAL_TRIALS research · 2025-08-22Clinical Trial Update: Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology [NA]
This clinical trial update describes the assessment of gait analysis parameters and upper-limb evaluation in adults with various neurologica…