日期 影响因子
日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:
–

Real-world clinical outcomes of patients with CLN2 disease treated with cerliponase alfa

接受塞利波酶α治疗的CLN2疾病患者的真实世界临床结果

期刊:Frontiers in Neurology
影响因子:2.8
doi:10.3389/fneur.2025.1516026
Schulz, Angela; Schwering, Christoph; Wibbeler, Eva; Westermann, Lena Marie; Hagenah, Luca; Lezius, Susanne; Jha, Ashok; Hunt, Abigail; Slasor, Peter; Reisewitz, Pascal; Nickel, Miriam
发表日期：2025
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Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data

探索CLN2临床评定量表的并发效度：使用cerliponase alfa临床试验数据与PedsQL进行比较

期刊:PLoS One
影响因子:2.6
doi:10.1371/journal.pone.0302382
Specchio, Nicola; Gissen, Paul; de Los Reyes, Emily; Olaye, Andrew; Camp, Charlotte; Curteis, Tristan; Griffiths, Annabel; Butt, Thomas; Cohen-Pfeffer, Jessica; Slasor, Peter; Sisic, Zlatko; Jain, Mohit; Schulz, Angela
发表日期：2024
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Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

使用 BMN 110（elosulfase alfa）进行酶替代疗法治疗 Morquio A 综合征（粘多糖贮积症 IVA）的疗效和安全性：一项 3 期随机安慰剂对照研究

期刊:Journal of Inherited Metabolic Disease
影响因子:3.8
doi:10.1007/s10545-014-9715-6
Hendriksz, Christian J; Burton, Barbara; Fleming, Thomas R; Harmatz, Paul; Hughes, Derralynn; Jones, Simon A; Lin, Shuan-Pei; Mengel, Eugen; Scarpa, Maurizio; Valayannopoulos, Vassili; Giugliani, Roberto; Slasor, Peter; Lounsbury, Debra; Dummer, Wolfgang
发表日期：2014
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