Overcoming obstacles in Parkinson's disease

Improved symptomatic and disease‐modifying treatments are needed for Parkinson's disease (PD). Although significant advances have been made in the understanding of PD etiology, the translation of these discoveries into novel transformative therapies has been limited as a result of systemic challenges in PD drug development. Preclinical testing lacks clear standards and prioritization criteria for advancing therapies to the clinic. Clinical testing is marked by expensive, long, and uninformative studies. In parallel to these scientific challenges, funding of late‐stage drug development has become increasingly scarce and risk averse. In this context, novel models of collaboration and funding are opening up new avenues for pursuing treatments. This review will discuss the most critical challenges in PD drug development and the innovative approaches being developed to overcome these hurdles. © 2012 Movement Disorder Society     

孕康糖浆治疗黄体功能不全性不孕患者临床观察

目的:观察孕康糖浆治疗黄体功能不全性不孕患者的临床疗效。方法:将84例黄体功能不全性不孕(肾虚型)患者按随机数字表法随机分为对照组和治疗组(n=42)。对照组予氯米芬治疗,治疗组加服孕康糖浆。测量基础体温、血清雌二醇(E2)、孕酮(P),B超观察排卵情况及子宫内膜厚度;进行黄体HPS评分,评价临床疗效。结果:治疗组临床总有效率85.7%,优于对照组;基础体温的高温相持续时间长于对照组;E2、P水平均高于对照组;优势卵泡最大直径、子宫内膜厚度和HPS评分均大于对照组(P<0.05或P<0.01)。结论:孕康糖浆对黄体功能不全性不孕具有肯定疗效,值得推广。     

The Wiskott–Aldrich syndrome: from genotype–phenotype correlation to treatment

Development of patient-tailored prognostic and therapeutic strategies remains a major, yet largely unmet, goal of medicine. Studies performed in patients who carry mutations in the Wiskott–Aldrich syndrome protein (WASP) gene have indicated the feasibility, but also the limitations, of this approach. In this review, we discuss the importance of specific WASP mutations, and their effects on protein expression and function in determining the spectrum of clinical phenotypes associated with WASP defects. The array of currently available and foreseeable therapeutic options is reviewed in this context as a model for other more common genetic disorders.     

A Patient with AKI after Cardiac Surgery

Up to 30% of patients undergoing cardiac surgery develop AKI, with 1% requiring RRT. AKI is an independent risk factor for morbidity and mortality. Postoperatively, even minimal changes in serum creatinine are associated with a substantial increase in mortality. No intervention has been definitely proven effective in reducing kidney injury. The successful prevention and management of AKI involves identifying patients at risk for AKI, recognizing subtle abnormalities in a timely manner, performing basic clinical assessments, and responding appropriately to data obtained. With that in mind, in this Attending Rounds, a woman with AKI in the setting of cardiac surgery is presented to highlight the use of history, physical exam, hemodynamic monitoring, laboratory data trends, and urine indices in establishing the correct diagnosis and appropriate management.