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후기 발병형 폼페병 환자의 평가측정도구 개발 : Modified Rasch-Built Pompe-Specific Activity scale

Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale
^{van Kooten HA, et al. Eur J Neurol. 2024:e16397}

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치료 경험이 없는 후기 발병형 폼페병 성인 환자에서 avalglucosidase alfa와 alglucosidase alfa 투여 후 치료 결과 비교: 3상 COMET 연구 사후분석

Effect of avalglucosidase alfa on disease-specific and general patient-reported outcomes in treatment- naïve adults with late-onset Pompe disease compared with alglucosidase alfa: Meaningful change analyses from the Phase 3 COMET trial
^{Toscano A, et al. Mol Genet Metab. 2024;141(2):108121.}

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프랑스의 폼페병 레지스트리를 활용한 후기 발병형 폼페병 환자의 bulbar muscle impairment 분석

Bulbar muscle impairment in patients with late onset Pompe disease: Insight from the French Pompe registry
^{Retailleau E, et al. Eur J Neurol. 2024;31(10):e16428.}

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