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Raptor Pharmaceuticals Doses First Patient In Phase 2b Clinical Trial Of DR Cysteamine For Cystinosis
Raptor Pharmaceuticals Corp. ("Raptor" or the "Company") (OTC Bulletin Board: RPTP), announced that it has dosed the first patient in its Phase 2b clinical trial, conducted in collaboration with the University of California, San Diego ("UCSD"), to evaluate Raptor"s proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") capsules in nephropathic cystinosis ("cystinosis"), a rare genetic lysosomal storage disease. Under Raptor"s open Investigational New Drug application ("IND"), UCSD is performing the Raptor-sponsored trial at its General Clinical Research Center.
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Teaching Families To Manage Asthma Can Reduce E.R. Visits

The key to reducing the leading cause of pediatric emergency room visits could be to educate young patients and their parents about how to manage asthma, according to an updated review of studies. Moreover, such programs could lead to fewer hospitalizations for children. The Cochrane review covered 38 randomized controlled trials involving 7,843 children. Studies looked at a variety of programs, with interventions delivered by medical professionals either in the emergency room, in the hospital, in homes or in community centers. Interventions included showing patients how to monitor their breathing flow, teaching them the importance of responding to early symptoms before an asthmatic reaction escalates into an emergency, and making environmental changes in the home, such as removing allergens and asthma triggers. Educational interventions were effective in reducing the risk of subsequent emergency room visits by just over 25 percent. Educational intervention also resulted in fewer unscheduled doctor visits. Among those delivering interventions were nurses or other health or medical practitioners either associated with a hospital or referred by a hospital. The review appears in the current issue of The Cochrane Library, a publication of The Cochrane Collaboration, an international organization that evaluates research in all aspects of health care. Systematic reviews draw evidence-based conclusions about medical practice after considering both the content and quality of existing trials on a topic. Toby Lasserson, review co-author at St. George"s, University of London, said that this was the second review of these studies. "We became aware of a number of trials coming through and we felt that in order for the review to be relevant to practice and policy, it needed to be updated to take account of the new evidence in this area." As the number of asthma-related emergency room visits increases, more hospitals are looking for effective methods to help prevent the expense and trauma of emergency treatment. "In addition to the high cost of treatment in a hospital setting, asthma emergencies can have a very disruptive effect on the quality of life for the child and their entire family," said Joseph Zorc, M.D., an associate professor of pediatrics and emergency medicine at The Children"s Hospital of Philadelphia. "Emergency visits are also a marker of poorly controlled chronic asthma that is a leading cause of childhood disability and missed school." Previous studies have confirmed that education can help prevent emergency room visits in adults and reviewers assumed they would see the same effect in children with asthma. "Asthma is a chronic disease," said Vincent Wang, M.D., associate director of the division of emergency and transport medicine at Children"s Hospital of Los Angeles. "Most patients who have more than intermittent symptoms require a controller medication and a rescue medication. With a treatment plan, asthma can be controlled and life-threatening exacerbations can be limited. If patients are being seen only when severe enough to warrant an emergency visit, patients are usually being under-treated." Most of the review studies focused on children under 10 years old. It is possible, the review authors say, that involving parents in administering maintenance therapies might help. It was harder to pinpoint specific results in adolescent patients, as they tended to drop out of studies at a higher rate. The authors concluded that educational interventions might particularly be useful for young patients who have a history of emergency room visits, as they might have more severe asthma and lack the skills to manage it effectively. The review suggests that if children and their parents receive an individualized asthma management plan, they could avoid many emergency department visits. Although the review recommended educational interventions, it was not clear which types of intervention were the most successful or what setting was preferred. The reviewers could draw no conclusions as to whether longer duration or greater intensity of the education delivered led to more outcomes that are successful. The studies did show that it was important to become cognizant of asthma triggers in the home such as smoking or using of mattress covers to reduce exposure to dust mites as part of a behavioral approach to asthma management. While the study did not demonstrate clear trends in effective education, Zorc did not find this surprising. "Given the heterogeneity of the studies and populations involved, it is likely that one size will not fit all. How the information is conveyed most effectively may vary by population and setting." The Cochrane Collaboration is an international nonprofit, independent organization that produces and disseminates systematic reviews of health care interventions and promotes the search for evidence in the form of clinical trials and other studies of interventions. Boyd M, et al. Interventions for educating children who are at risk for asthma-related emergency department attendance. Cochrane Database of Systematic Reviews 2009, Issue 2. Health Behavior News Service


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