JIMD reports

Long-Term Safety and Efficacy of Pegvaliase in Japanese Adults With Phenylketonuria: Final Results of a Phase III Trial.
日本苯丙酮尿症成人中 Pegvisomase 的长期安全性与有效性 :III 期试验的最终结果。

Unifying the Communities of Early-Onset Glycogen Storage Disease Type IV and Adult Polyglucosan Body Disease Through a Genetic Prevalence Study of GBE1-Related Disease.
通过 GBE1 相关疾病的遗传患病率研究，统一早发性糖原贮积病 IV 型和成人多糖体病的社区。

Alkaline Phosphatase and Infantile GM1 Gangliosidosis: A Simple Biomarker for a Complex Disease?
碱性磷酸酶和婴儿 GM1 神经节苷脂贮积症:复杂疾病的简单生物标志物?