A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B
一项针对B型圣菲利波综合征患者的脑室内注射替拉西尼酶α的I/II期研究
期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI165076
Muschol, Nicole; Koehn, Anja; von Cossel, Katharina; Okur, Ilyas; Ezgu, Fatih; Harmatz, Paul; de Castro Lopez, Maria J; Couce, Maria Luz; Lin, Shuan-Pei; Batzios, Spyros; Cleary, Maureen; Solano, Martha; Nestrasil, Igor; Kaufman, Brian; Shaywitz, Adam J; Maricich, Stephen M; Kuca, Bernice; Kovalchin, Joseph; Zanelli, Eric