Records containing: Pompe Disease
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-04-22A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Investigate the Safety, Pharmacodynamics, and Preliminary Efficacy of S-606001 as an Add-on to Enzyme Replacement Therapy in Patients With Late-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2026-04-13A Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2026-04-09Pompe Disease Registry
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CLINICAL_TRIALS study record update · 2026-04-08Determination of Cross-Reactive Immunological Material (CRIM) Status and Longitudinal Follow-up of Individuals With Pompe Disease
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CLINICAL_TRIALS study record update · 2026-03-10A Global Prospective Observational Registry of Patients With Pompe Disease
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CLINICAL_TRIALS study record update · 2026-03-04Cognitive and Neurological Pathologies in Pompe Disease
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CLINICAL_TRIALS study record update · 2026-01-16A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme/Nexviadyme (Avalglucosidase Alfa-ngpt/Avalglucosidase Alfa) During Pregnancy and/or Lactation in the Postmarketing Setting
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CLINICAL_TRIALS study record update · 2025-10-23An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease
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CLINICAL_TRIALS study record update · 2025-09-22Developing a Management Approach for Patients With "Late-Onset" Pompe Disease GAA Variant Identified by Newborn Screening
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CLINICAL_TRIALS study record update · 2025-09-15Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease
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CLINICAL_TRIALS study record update · 2025-09-10Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
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CLINICAL_TRIALS study record update · 2025-08-26Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive
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CLINICAL_TRIALS study record update · 2025-08-12Universal Genomic Newborn Screening in the Wallonia-Brussels Federation: Baby Detect
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CLINICAL_TRIALS study record update · 2025-03-12Multispectral Optoacoustic Tomography for Translational Molecular Imaging in Pompe Disease
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CLINICAL_TRIALS study record update · 2024-11-27Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2024-11-14FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
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CLINICAL_TRIALS study record update · 2024-06-24Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
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CLINICAL_TRIALS study record update · 2024-03-19A Sub-Registry to Determine the Presence of Alglucosidase Alfa in Breast Milk From Women With Pompe Disease Treated With Alglucosidase Alfa.
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CLINICAL_TRIALS study record update · 2024-02-14Multi-Centre, Non-Interventional, Double Cohort Study to Assess the Safety of Myozyme® and of Aldurazyme® in Real-World Home Infusion Setting
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CLINICAL_TRIALS study record update · 2023-12-05Development and Validation of a Standardized Assessment Instrument for Health-related Quality of Life (HrQoL) in Children and Adolescents With Pompe Disease (PD): a Joint Effort of Patients, Caregivers and Metabolic Experts
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CLINICAL_TRIALS study record update · 2023-11-14Multi-ingredient Supplementation as an Adjunctive Therapy in Late-onset Pompe Disease
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CLINICAL_TRIALS study record update · 2023-06-05Response to Diaphragmatic Pacing in Subjects With Pompe Disease
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CLINICAL_TRIALS study record update · 2023-05-30The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease
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CLINICAL_TRIALS study record update · 2023-04-19National Taiwan University Hospital
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CLINICAL_TRIALS study record update · 2023-03-24A Multi-Center, Low-Interventional Study With a Retrospective Component in Participants With Late-Onset Pompe Disease