Beacon Clinical Research

<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto" id="d16520194e83">Drug repurposing has huge potential to quickly drive therapeutic hypotheses to the clinic and deliver patient impact. This alone makes repurposing an attractive strategy for rare diseases, where there is huge unmet medical need caused by a paucity of treatment options and scientific understanding. Identifying repurposing opportunities in such an understudied environment is challenging, while recruiting and retaining a significant number of patients for clinical trials can be prohibitive. In this environment, patient engagement is central to repurposing success. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d16520194e85">The REMEDi4ALL platform has been developed to have patient input at the heart of its repurposing projects for this very reason. REMEDi4ALL aims to include a patient champion in the heart of our repurposing development teams. These individuals provide vital patient input, guiding project development to ensure a focus on the expressed needs of the patient community, ensuring meaningful impact and real value to society. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d16520194e87">The best example of the impact of this model comes from REMEDi4ALL’s demonstrator project to repurpose tazarotene for the ultrarare multisulfatase deficiency. Working alongside REMEDi4ALL’s patient champion and the United MSD foundation has unlocked a pharmaceutical industry data package to support our project. Furthermore, we have gathered detailed patient perspective data to influence the scientific advice process, answering vital questions from the regulators through patient experience. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d16520194e89">Here, we review the benefits and challenges of REMEDi4ALL’s approach, its impact on our demonstrator project, and our hopes for patient engagement in future repurposing projects.

Abstract MTBE is a colorless, relatively volatile liquid that has found widespread use as an octane‐enhancing gasoline additive. In 1987, the Environmental Protection Agency's (EPA) Interagency Testing Committee identified MTBE for priority testing consideration based on large production volume, potential widespread exposure, and limited data on chronic health effects. In response, the industry formed the MTBE Health Effects Testing Task Force, which in 1988 signed a Consent Agreement with the EPA requiring the task force member companies to perform toxicological testing on MTBE. The testing program, which began in the second quarter of 1988, consists of a full complement of short‐ and long‐term tests. The testing completed to date includes genotoxicity (in vivo bone marrow cytogenetics and Drosophila sex‐linked recessive lethal assays), developmental toxicity, acute and subchronic neurotoxicity (motor activity, functional observation battery, and neuropathology), subchronic toxicity, reproductive/fertility effects, and pharmacokinetic studies. There is also an ongoing oncogenicity study in rats and mice. The final report for this chronic study is expected at the end of 1992. The total cost for the program is approximately $3.75 million, which is funded by the 11 Task Force member companies based on market share. These studies were sponsored by the MTBE Health Effects Testing Task Force, Oxygenated Fuels Association, Washington, D.C. Key words: MTBEhealth effectstoxicityexposure

&lt;div class="htmlview paragraph"&gt;A 215 car “consumer type” road test was run to investigate the influence of various motor oils on fuel economy. Under “real life” conditions, the differences between conventional 10W-40 oils and several 10W-40 oils formulated for fuel economy were small. Furthermore, such differences would be difficult for the individual consumer to detect as a result of other variables such as changes in driving patterns and the effect of seasonal temperature changes. The methods for adjusting the data to compensate for these factors are explained.&lt;/div&gt;

e) At the lower sliding .speeds the film thickness becomes so small that boundary processes must lake over before the collision is complete.

The LaryngoscopeVolume 47, Issue 6 p. 418-420 Article Symposium: Neural mechanism of hearing: II.—Physiology. (c)—Pathology of the organ of corti† Dr. M. H. Lurie, Dr. M. H. Lurie 483 Beacon Street, Boston.Search for more papers by this author Dr. M. H. Lurie, Dr. M. H. Lurie 483 Beacon Street, Boston.Search for more papers by this author First published: June 1937 https://doi.org/10.1288/00005537-193706000-00007 † Read as part of the Symposium, “The Neural Mechanism of Hearing,” at the Seventieth Annual Meeting of the American Otological Society, Long Beach, N. Y., May 27, 1937 AboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onFacebookTwitterLinkedInRedditWechat Volume47, Issue6June 1937Pages 418-420 RelatedInformation