Does early treatment of PKU patients with sapropterin dihydrochloride affect brain development?
收藏Alliance of Genome Resources2026-08-01 收录
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We show deleterious effects of SD on developing brain cells in a rat in vitro model. This observation raises the question whether the use of SD can be recommended in very young PKU patients as currently licensed.
本研究在大鼠体外模型中证实了SD对发育中脑细胞的有害作用。这一观察结果提出了一个疑问:按照当前获批的用法,SD是否可推荐用于极年幼的苯丙酮尿症(Phenylketonuria)患者?




