Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome)
Juyoung Sung, Insung Kim, Minji Im, Yoon Ji Ahn, Sangmi Kim, Ja‐Hyun Jang, Hyung‐Doo Park, Tae Yeon Jeon, Kyung Rae Ko, Se‐Jun Park, Jun Hwa Lee, Eun Young Kim, Chong Kun Cheon, Eungu Kang, Jung-Eun Moon, Young Bae Sohn, Hsiang‐Yu Lin, Chih‐Kuang Chuang, Shuan-Pei Lin, Sung Yoon Cho
IF 1.9 (2025) Molecular Genetics and Metabolism Reports
초록
ERT로 치료하였으며, 새롭게 확인된 안전성 우려는 없었다.
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