Records containing: Pompe Disease Infantile-Onset
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 · 2026-04-08Registry of Patients Diagnosed With Lysosomal Storage Diseases
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CLINICAL_TRIALS study record update · 2026-03-17PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
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CLINICAL_TRIALS study record update · 2025-09-10Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease
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CLINICAL_TRIALS study record update · 2025-07-03A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease
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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 · 2021-02-16Acute and Long Term Evaluation of Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.
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CLINICAL_TRIALS study record update · 2014-02-05A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies