|By Business Wire||
|May 14, 2014 08:09 AM EDT||
MyoKardia, Inc., a company pioneering the development of precision therapies for genetic heart disease, today announced the launch of the Sarcomeric Human Cardiomyopathy Registry (SHaRe), a multi-center, international repository of clinical data on individuals with genetic heart disease. The registry was developed in collaboration with investigators from seven world-leading cardiovascular centers and currently contains de-identified data on over 5,800 subjects. SHaRe aims to grow into the world’s most robust database of clinical information on genetic heart disease through relationships with additional clinical investigators and cardiovascular centers of excellence.
SHaRe is a first-of-its-kind effort that promises to yield a deeper understanding of genetic heart disease and pave the way for more targeted and effective approaches to treating the disease. Two types of genetic heart disease, hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM), together affect nearly 800,000 people in the United States, but treatment to halt the progression of disease has remained elusive due to the complex and varied nature of these conditions. Mutations in the molecular motor of the heart, the sarcomere, are an important cause of these inherited heart muscle disorders. Patients with HCM and DCM do not all share the same disease-causing genetic mutations, or genotype. There are many different clinical characteristics, or phenotypes, in part driven by their individual genetic makeup. SHaRe will play an important role in helping researchers and clinicians better understand how these genotypes and phenotypes interact, which will ultimately lead to the development of targeted therapies based on the specific disease-causing genetic mutations in individual patients.
“Genetic heart disease can present itself in both children and adults. Moreover, the burden of symptoms and the severity of disease are highly variable,” said Carolyn Ho, M.D., assistant professor of medicine at Harvard Medical School, medical director of the Cardiovascular Genetics Center at Brigham and Women’s Hospital and lead SHaRe investigator. “Our goal in creating SHaRe is to critically advance the understanding of HCM and DCM. By amassing these detailed longitudinal and centralized data, we hope to generate better predictors of risk and disease progression. With this information, we can better understand why some patients do poorly, and as importantly, why some patients do well. By redefining and refining phenotypes and modifiers of outcome in genetic heart disease, we ultimately hope to improve the care of our patients and families.”
The centers of excellence currently participating in SHaRe include Brigham and Women’s Hospital, Boston; University of Michigan Medical Center; Stanford University Medical Center; Boston Children’s Hospital; The Heart Hospital, University College London, U.K.; Florence Centre for Cardiomyopathies, Italy; and Erasmus University Medical Center, Netherlands.
Despite the often devastating effect on families, public awareness and understanding of genetic heart disease remains low. As part of SHaRe, a companion website has been launched to provide patients, families and communities with information about heritable cardiomyopathies, SHaRe centers and investigators, related research activities and other genetic heart disease information.
“Together with our SHaRe collaborators, MyoKardia is committed to helping patients with genetic heart disease and their families,” said Jonathan C. Fox, M.D., Ph.D., chief medical officer of MyoKardia and consulting professor at the Stanford Cardiovascular Institute. “Currently available treatment options for patients with heritable cardiomyopathies are limited and do not target the primary cause of the disease. For these patients, a ‘one-size-fits-all’ approach to therapy simply won’t work. The insights gleaned from this large database of clinical information will allow us to better tailor our programs in HCM and DCM and advance our goal of enabling precision medicines for patients with genetic heart disease.”
MyoKardia is developing a pipeline of novel, small-molecule targeted therapeutics to treat genetic heart disease based on mutations in heart muscle that are known to cause the disease. The company’s current programs are focused on both HCM and DCM, for which no therapeutics have ever been approved.
About Genetic Heart Disease
Hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM) are types of genetic heart disease that are caused by mutations in the protein genes of the sarcomere, the fundamental contractile unit of heart muscle, and passed on in families in an autosomal dominant pattern of inheritance. HCM impacts an estimated one in 500 people in the general population by producing thickening of the heart walls and is recognized as a cause of sudden cardiac death, the most common cause of non-traumatic death in young adults. DCM produces weakening of the heart walls and enlargement of the heart chambers and is estimated to occur in one in 2,500 people in the general population. The prevalence of HCM and DCM, taken together, is estimated at nearly 800,000 people in the United States, affecting both children and adults.
MyoKardia, Inc. is dedicated to revolutionizing the treatment of genetic heart diseases through the development of novel, small-molecule targeted therapeutics that address the underlying cause of disease. By combining leading-edge cardiovascular genetics with recent advances in heart muscle biochemistry, MyoKardia seeks to usher in an era of precision medicine that will dramatically improve the treatment of cardiomyopathies and make a meaningful difference in the lives of people with genetic heart disease. Launched in 2012 by a group of researchers widely acknowledged as the world leaders in their fields, MyoKardia is funded by leading life sciences investor Third Rock Ventures. For more information, please visit www.myokardia.com.
Recognizing the need to identify and validate information security professionals’ competency in securing cloud services, the two leading membership organizations focused on cloud and information security, the Cloud Security Alliance (CSA) and (ISC)^2, joined together to develop an international cloud security credential that reflects the most current and comprehensive best practices for securing and optimizing cloud computing environments.
Feb. 13, 2016 08:00 AM EST
DevOps is not just last year’s buzzword. Companies with DevOps practices are 2.5x more likely to exceed profitability, market share, and productivity goals. But how do you enable high performance? What can you do right now to start? Find out from DevOps experts including Gene Kim, co-author of "The Phoenix Project," and the Dynatrace Center of Excellence.
Feb. 13, 2016 07:30 AM EST
Predictive analytics tools monitor, report, and troubleshoot in order to make proactive decisions about the health, performance, and utilization of storage. Most enterprises combine cloud and on-premise storage, resulting in blended environments of physical, virtual, cloud, and other platforms, which justifies more sophisticated storage analytics. In his session at 18th Cloud Expo, Peter McCallum, Vice President of Datacenter Solutions at FalconStor, will discuss using predictive analytics to ...
Feb. 13, 2016 06:45 AM EST Reads: 418
Silver Spring Networks, Inc. (NYSE: SSNI) extended its Internet of Things technology platform with performance enhancements to Gen5 – its fifth generation critical infrastructure networking platform. Already delivering nearly 23 million devices on five continents as one of the leading networking providers in the market, Silver Spring announced it is doubling the maximum speed of its Gen5 network to up to 2.4 Mbps, increasing computational performance by 10x, supporting simultaneous mesh communic...
Feb. 13, 2016 05:00 AM EST
Eighty percent of a data scientist’s time is spent gathering and cleaning up data, and 80% of all data is unstructured and almost never analyzed. Cognitive computing, in combination with Big Data, is changing the equation by creating data reservoirs and using natural language processing to enable analysis of unstructured data sources. This is impacting every aspect of the analytics profession from how data is mined (and by whom) to how it is delivered. This is not some futuristic vision: it's ha...
Feb. 13, 2016 04:45 AM EST Reads: 466
The cloud promises new levels of agility and cost-savings for Big Data, data warehousing and analytics. But it’s challenging to understand all the options – from IaaS and PaaS to newer services like HaaS (Hadoop as a Service) and BDaaS (Big Data as a Service). In her session at @BigDataExpo at @ThingsExpo, Hannah Smalltree, a director at Cazena, will provide an educational overview of emerging “as-a-service” options for Big Data in the cloud. This is critical background for IT and data profes...
Feb. 13, 2016 03:45 AM EST Reads: 244
Father business cycles and digital consumers are forcing enterprises to respond faster to customer needs and competitive demands. Successful integration of DevOps and Agile development will be key for business success in today’s digital economy. In his session at DevOps Summit, Pradeep Prabhu, Co-Founder & CEO of Cloudmunch, covered the critical practices that enterprises should consider to seamlessly integrate Agile and DevOps processes, barriers to implementing this in the enterprise, and pr...
Feb. 13, 2016 03:00 AM EST Reads: 466
Sensors and effectors of IoT are solving problems in new ways, but small businesses have been slow to join the quantified world. They’ll need information from IoT using applications as varied as the businesses themselves. In his session at @ThingsExpo, Roger Meike, Distinguished Engineer, Director of Technology Innovation at Intuit, showed how IoT manufacturers can use open standards, public APIs and custom apps to enable the Quantified Small Business. He used a Raspberry Pi to connect sensors...
Feb. 13, 2016 02:30 AM EST Reads: 377
The principles behind DevOps are not new - for decades people have been automating system administration and decreasing the time to deploy apps and perform other management tasks. However, only recently did we see the tools and the will necessary to share the benefits and power of automation with a wider circle of people. In his session at DevOps Summit, Bernard Sanders, Chief Technology Officer at CloudBolt Software, explored the latest tools including Puppet, Chef, Docker, and CMPs needed to...
Feb. 13, 2016 02:30 AM EST Reads: 369
Let’s face it, embracing new storage technologies, capabilities and upgrading to new hardware often adds complexity and increases costs. In his session at 18th Cloud Expo, Seth Oxenhorn, Vice President of Business Development & Alliances at FalconStor, will discuss how a truly heterogeneous software-defined storage approach can add value to legacy platforms and heterogeneous environments. The result reduces complexity, significantly lowers cost, and provides IT organizations with improved effi...
Feb. 13, 2016 12:45 AM EST Reads: 281
It's easy to assume that your app will run on a fast and reliable network. The reality for your app's users, though, is often a slow, unreliable network with spotty coverage. What happens when the network doesn't work, or when the device is in airplane mode? You get unhappy, frustrated users. An offline-first app is an app that works, without error, when there is no network connection.
Feb. 12, 2016 10:00 PM EST Reads: 247
Data-as-a-Service is the complete package for the transformation of raw data into meaningful data assets and the delivery of those data assets. In her session at 18th Cloud Expo, Lakshmi Randall, an industry expert, analyst and strategist, will address: What is DaaS (Data-as-a-Service)? Challenges addressed by DaaS Vendors that are enabling DaaS Architecture options for DaaS
Feb. 12, 2016 09:45 PM EST Reads: 388
One of the bewildering things about DevOps is integrating the massive toolchain including the dozens of new tools that seem to crop up every year. Part of DevOps is Continuous Delivery and having a complex toolchain can add additional integration and setup to your developer environment. In his session at @DevOpsSummit at 18th Cloud Expo, Miko Matsumura, Chief Marketing Officer of Gradle Inc., will discuss which tools to use in a developer stack, how to provision the toolchain to minimize onboa...
Feb. 12, 2016 09:00 PM EST Reads: 136
Companies can harness IoT and predictive analytics to sustain business continuity; predict and manage site performance during emergencies; minimize expensive reactive maintenance; and forecast equipment and maintenance budgets and expenditures. Providing cost-effective, uninterrupted service is challenging, particularly for organizations with geographically dispersed operations.
Feb. 12, 2016 06:00 PM EST
SYS-CON Events announced today that Catchpoint Systems, Inc., a provider of innovative web and infrastructure monitoring solutions, has been named “Silver Sponsor” of SYS-CON's DevOps Summit at 18th Cloud Expo New York, which will take place June 7-9, 2016, at the Javits Center in New York City, NY. Catchpoint is a leading Digital Performance Analytics company that provides unparalleled insight into customer-critical services to help consistently deliver an amazing customer experience. Designed...
Feb. 12, 2016 06:00 PM EST Reads: 406