Limited capacity in US pediatric drug trials: qualitative analysis of expert interviews [0.03%]
美国儿科临床试验供药量受限问题：专家访谈定性分析
Richard Wasserman,Alison Bocian,Donna Harris et al.
Richard Wasserman et al.
Background: The recently renewed Best Pharmaceuticals for Children and Pediatric Research Equity Acts (BPCA/PREA) have continued industry incentives and opportunities for pediatric drug trials (PDTs). However, there is no...
Patricia K Crumrine
Patricia K Crumrine
Lennox-Gastaut syndrome is an epilepsy syndrome that begins in childhood (between 1 and 8 years of age), worsens during latency and persists frequently into adulthood, is refractory to antiepileptic medications, and results in cognitive dec...
Rufinamide for pediatric patients with Lennox-Gastaut syndrome: a comprehensive overview [0.03%]
鲁非尼胺治疗兰诺克斯-加斯特综合征患者的疗效及安全性：综述文章
Heather Ann Wier,Ana Cerna,Tsz-Yin So
Heather Ann Wier
Rufinamide is a triazole derivative with broad-spectrum antiepileptic effects that is unrelated to any antiepileptic drug currently on the market. The European Commission and the US FDA approved rufinamide in 2007 and 2008, respectively, fo...
Samar H Ibrahim,Keith D Lindor
Samar H Ibrahim
Primary sclerosing cholangitis (PSC) is a chronic cholestatic condition affecting the extrahepatic and intrahepatic biliary tree. The incidence is seemingly increasing in children as a result of increased use of cholangiographic screening t...
Multisystem Langerhans cell histiocytosis in children: current treatment and future directions [0.03%]
儿童多系统朗格汉斯细胞组织细胞增生症的当前治疗及未来方向
Milen Minkov
Milen Minkov
Langerhans cell histiocytosis (LCH) is a rare (about 3-5 cases per million children aged 0-14 years), non-malignant disease characterized by proliferation and accumulation of clonal dendritic cells, extreme clinical heterogeneity, and an un...
Kristina Star
Kristina Star
Calcineurin inhibitor-free immunosuppression in pediatric renal transplantation: a viable option? [0.03%]
儿童肾移植中无需钙调神经磷酸酶抑制剂的免疫抑制方案可靠吗？
Britta Höcker,Burkhard Tönshoff
Britta Höcker
The introduction, in the mid-1980s, of calcineurin inhibitors - namely ciclosporin (cyclosporine) and later tacrolimus - has significantly improved short-term renal graft survival by lowering acute rejection rates in both adult and pediatri...
Disease registries and outcomes research in children: focus on lysosomal storage disorders [0.03%]
儿童疾病登记和结果研究：以溶酶体贮积病为重点
Simon Jones,Emma James,Suyash Prasad
Simon Jones
Assessing medicines specifically for use in children has been neglected in the past, with the majority of formal clinical studies being conducted in adults. Clinical trials are a pivotal part of the drug approval process; however, they are ...