IMAGE:This image shows the cerebellum of a brain affected by Niemann-Pick disease type C at the end stage of the disease. The blue staining shows the dense pockets of... view more Credit: National Institutes of Health, NIH/Eunice Kennedy Shriver National Institute of Child Health and Human Development Researchers from the National Institutes of Health have entered into an agreement with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal storage disorders. Lysosomal storage diseases, also known as lipid storage diseases, comprise about 50 rare inherited disorders that usually affect children. Fatty materials accumulate in the cells and tissues of the body. These diseases can result in damage to the brain, peripheral nervous system, liver, and other organs and tissues; they are often fatal. Researchers at the National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), both parts of NIH, will conduct studies on NPC and other lysosomal storage disorders with funding provided by Vtesse. "This is an excellent example of how launching a project to study the underlying biology of one disease can lead to advances that hold … Continue reading →

Listen Story audio 37min 24sec Susan Wolf: Professor of law, medicine and public policy at the University of Minnesota Robert Green: Medical geneticist at Harvard Medical School and Brigham & Women's Hospital Genetic tests are giving patients unprecedented insights into conditions that they could inherit, but should this information be made available to other family members who might also be susceptible? On The Daily Circuit, we discuss the tension between awareness and privacy in genetic testing. Susan Wolf, professor of law, medicine and public policy at the University of Minnesota joins the discussion along with Dr. Robert Green, a medical geneticist at Harvard Medical School. From Science Friday: That's even true when information can be vital for for the health of family members. "Let's say a researcher is doing a genetic study ... and they discover that their participant has a variant of the BRCA gene, associated with higher risk of breast and ovarian cancer," says Susan M. Wolf, the McKnight Presidential Professor of Law, Medicine and Public Policy at the University of Minnesota. "They go to that person and say, 'We think it's important for you to know this, but we're also concerned about your sister, or your brother, … Continue reading →

Published on January 7th, 2015 | by LedgerOnline By Diana Burmistrovich/JNS.org One in four Jews is a carrier of one or more of the 19 known preventable Jewish genetic diseases, according to the Center for Jewish Genetics. Although Sephardic Jews and non-Jews can carry these diseases, they appear twice as often for Ashkenazi Jews as they do for the rest of the population. When both spouses are carriers for a particular genetic disease, the couple has a 25 percent chance of passing the disease on to their children. Launched in September through the Emory University School of Medicines Department of Human Genetics, the goal of the JScreen not-for-profit health initiative is to make those statistics appear less daunting. A carrier-screening program for Jewish genetic diseases, JScreen aims to give families with Jewish ancestry easy access to information and to provide convenient testing. Employing an easy-to-use kit, JScreen allows individuals to test for the 19 known preventable Jewish genetic diseaseswhich among others include Tay-Sachs, Canavan, and Gaucherin their own homes. While testing for genetic disorders is nothing new, JScreens accessibility is. The kit is easily acquired through the initiatives website atwww.JScreen.org, and the test allows a saliva sample to be sentdirectly … Continue reading →

PARIS--(BUSINESS WIRE)--Regulatory News: NANOBIOTIX (Paris:NANO) (Euronext: NANO ISIN: FR0011341205), a late clinical-stage nanomedicine company pioneering novel approaches for the local treatment of cancer, provides its activities and achievements during 2014 and an overview of anticipated 2015 events including the financial calendar. The Company made major clinical and financial progress, including broadening of indications and strategic plans across Europe, Asia and the US. Highlights Laurent Levy, CEO of Nanobiotix said: 2014 has been a pivotal year for the Company, with significant progress in clinical development, where we jumped from an early clinical stage to late clinical stage. This has impacted our visibility and exposure from a financial and industrial point of view allowing us to accomplish two capital raises this year. We are now in a position to create value through the expansion of the indications and geographical area of development for the lead product, NBTXR3. As a result, 2015 is expected to be a year of further significant progress with intermediary clinical results, US corporate developments and launch of new indications; all building on the successes of 2014 and preparing for the final steps of CE marking by the end 2016 required before commercial launch. Overview Expansion of clinical development … Continue reading →

SAN FRANCISCO (MarketWatch) Shares of WD-40 Co. slipped in the extended session Wednesday on an earnings miss. WD-40 WDFC, +2.99% shares fell 5.2% to $81 on light volume after the companys fiscal first-quarter results fell short of Wall Street expectations. Shares of NephroGenex Inc. NRX, -0.75% rose 19% to $10.99 on moderate volume after the clinical-stage drug company said late-stage studies on its diabetic nephropathy treatment Pyridorin would be able to support a marketing application in Europe. The company also disclosed that RHO Capital had taken an 11.9% stake in the company. Bind Therapeutics Inc. BIND, -4.28% shares jumped 26% to $6.50 on moderate volume. The nanomedicine platform company said it expects one of its collaboration partners will file a drug application with the FDA by mid-2015 using their Accurin drug delivery particles. Mistras Group Inc. MG, +3.51% shares rose 13% to $20.63 on light volume after the company reported fiscal second-quarter earnings of 33 cents a share on revenue of $206.9 million. Analysts had estimated 26 cents a share on revenue of $186.2 million. Alexion Pharmaceuticals Inc. ALXN, +5.61% shares fell 4.8% to $179 in late trading after the company said a phase 2 clinical trial of eculizumab -- … Continue reading →