Lv22
180 积分 2021-06-23 加入
Mixed‐effects models for slope‐based endpoints in clinical trials of chronic kidney disease
1天前
已完结
Urinary globotriaosylceramide excretion correlates with the genotype in children and adults with Fabry disease
1天前
已完结
Elevation of urinary globotriaosylceramide (GL3) in infants with Fabry disease
1天前
已完结
The potential of gene editing for Huntington’s disease
9天前
已完结
Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy
18天前
已完结
Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2–neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial
20天前
已完结
Interpretation of GFR slope in untreated and treated adult Fabry patients
24天前
已完结
Gene Editing for CEP290 -Associated Retinal Degeneration
29天前
已完结
Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
29天前
已完结
Next-generation CRISPR-based gene-editing therapies tested in clinical trials
30天前
已完结
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