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| Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease |
| Chong Kun Cheon |
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Clin Exp Pediatr. 2020;63(1):14-15. Published online August 7, 2019 DOI: https://doi.org/10.3345/kjp.2018.07402 |
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Considerations for evaluating the effectiveness and long-term outcome of enzyme replacement therapy in Pompe disease Clinical and Experimental Pediatrics. 2020;63(1):14-15  Long-Term Interruption of Enzyme Replacement Therapy with rhGAA in Pompe Disease Leads to Irreversible Clinical Decline Journal of Neuromuscular Diseases. 2015;2(s1):S20-S20 Unwarranted, long term, alglucosidase alfa enzyme replacement therapy in two non-Pompe disease patients Clinical Neurology and Neurosurgery. 2020;196:106048 Pregnancy outcome in a Pompe disease patient treated with enzyme replacement therapy Clinical Therapeutics. 2010;32:S52 Long-term effects of enzyme replacement therapy in an elderly cohort of late-onset Pompe disease Neuromuscular Disorders. 2022;32(3):195-205 900. Desensitization through Immunomodulatory Gene Therapy Suppresses the Antibody Response to Enzyme Replacement Therapy in Murine Pompe Disease Molecular Therapy. 2009;17:S344 Effect of long term enzyme replacement therapy in late onset Pompe disease: A single-centre experience Neuromuscular Disorders. 2021;31(2):91-100 Respiratory function in late-onset Pompe disease patients receiving long-term enzyme replacement therapy for more than 48 months Wiener Medizinische Wochenschrift. 2012;163(1-2):40-44 Long-term enzyme-replacement therapy (ERT) with alglucosidase alfa: Evolution of two siblings with juvenile late-onset Pompe disease Journal of the Neurological Sciences. 2015;358(1-2):459-460 Long term enzyme replacement therapy for Fabry disease: effectiveness on heart, kidney and brain Molecular Genetics and Metabolism. 2017;120(1-2):S33 |
