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Clinical and molecular characterization of Korean children with infantile and late-onset Pompe disease: 10 years of experience with enzyme replacement therapy at a single center |
Min-Sun Kim, Ari Song, Minji Im, June Huh, I-Seok Kang, Jinyoung Song, Aram Yang, Jinsup Kim, Eun-Kyung Kwon, Eu-Jin Choi, Sun-Ju Han, Hyung-Doo Park, Sung Yoon Cho, Dong-Kyu Jin |
Clin Exp Pediatr. 2019;62(6):224-234. Published online October 4, 2018 DOI: https://doi.org/10.3345/kjp.2018.06968 |
Clinical and molecular characterization of Korean children with infantile and late-onset Pompe disease: 10 years of experience with enzyme replacement therapy at a single center Clinical improvement in a case of atypical infantile onset Pompe disease with enzyme replacement therapy Enzyme replacement therapy rate escalation in infantile onset Pompe disease Weekly enzyme replacement therapy: a French patient with infantile onset Pompe disease Effects of pre-symptomatic initiation of enzyme replacement therapy for infantile onset Pompe disease Enzyme replacement therapy and antibodies in late-onset Pompe disease T.P.43 Enzyme replacement therapy in late-onset Pompe disease – A systematic literature review Clinique : Late-onset Pompe disease: a new molecule for enzyme replacement therapy FIVE-YEARS FOLLOW-UP OF THE RESPIRATORY AND MOTOR INVOLVEMENTS IN PATIENTS WITH LATE ONSET POMPE DISEASE (LOPD) ON IRREGULAR ENZYME REPLACEMENT THERAPY (ERT) Effects of enzyme replacement therapy on bone density in late onset Pompe disease |