|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.
DevOps is often described as a combination of technology and culture. Without both, DevOps isn't complete. However, applying the culture to outdated technology is a recipe for disaster; as response times grow and connections between teams are delayed by technology, the culture will die. A Nutanix Enterprise Cloud has many benefits that provide the needed base for a true DevOps paradigm.
Mar. 25, 2017 12:00 AM EDT Reads: 1,508
What sort of WebRTC based applications can we expect to see over the next year and beyond? One way to predict development trends is to see what sorts of applications startups are building. In his session at @ThingsExpo, Arin Sime, founder of WebRTC.ventures, will discuss the current and likely future trends in WebRTC application development based on real requests for custom applications from real customers, as well as other public sources of information,
Mar. 25, 2017 12:00 AM EDT Reads: 403
SYS-CON Events announced today that Interoute, owner-operator of one of Europe's largest networks and a global cloud services platform, has been named “Bronze Sponsor” of SYS-CON's 20th Cloud Expo, which will take place on June 6-8, 2017 at the Javits Center in New York, New York. Interoute is the owner-operator of one of Europe's largest networks and a global cloud services platform which encompasses 12 data centers, 14 virtual data centers and 31 colocation centers, with connections to 195 add...
Mar. 24, 2017 11:45 PM EDT Reads: 581
Historically, some banking activities such as trading have been relying heavily on analytics and cutting edge algorithmic tools. The coming of age of powerful data analytics solutions combined with the development of intelligent algorithms have created new opportunities for financial institutions. In his session at 20th Cloud Expo, Sebastien Meunier, Head of Digital for North America at Chappuis Halder & Co., will discuss how these tools can be leveraged to develop a lasting competitive advanta...
Mar. 24, 2017 11:15 PM EDT Reads: 2,479
TechTarget storage websites are the best online information resource for news, tips and expert advice for the storage, backup and disaster recovery markets. By creating abundant, high-quality editorial content across more than 140 highly targeted technology-specific websites, TechTarget attracts and nurtures communities of technology buyers researching their companies' information technology needs. By understanding these buyers' content consumption behaviors, TechTarget creates the purchase inte...
Mar. 24, 2017 10:15 PM EDT Reads: 4,131
With the introduction of IoT and Smart Living in every aspect of our lives, one question has become relevant: What are the security implications? To answer this, first we have to look and explore the security models of the technologies that IoT is founded upon. In his session at @ThingsExpo, Nevi Kaja, a Research Engineer at Ford Motor Company, will discuss some of the security challenges of the IoT infrastructure and relate how these aspects impact Smart Living. The material will be delivered i...
Mar. 24, 2017 10:00 PM EDT Reads: 1,865
In his session at @ThingsExpo, Eric Lachapelle, CEO of the Professional Evaluation and Certification Board (PECB), will provide an overview of various initiatives to certifiy the security of connected devices and future trends in ensuring public trust of IoT. Eric Lachapelle is the Chief Executive Officer of the Professional Evaluation and Certification Board (PECB), an international certification body. His role is to help companies and individuals to achieve professional, accredited and worldw...
Mar. 24, 2017 08:15 PM EDT Reads: 276
My team embarked on building a data lake for our sales and marketing data to better understand customer journeys. This required building a hybrid data pipeline to connect our cloud CRM with the new Hadoop Data Lake. One challenge is that IT was not in a position to provide support until we proved value and marketing did not have the experience, so we embarked on the journey ourselves within the product marketing team for our line of business within Progress. In his session at @BigDataExpo, Sum...
Mar. 24, 2017 06:30 PM EDT Reads: 2,610
Your homes and cars can be automated and self-serviced. Why can't your storage? From simply asking questions to analyze and troubleshoot your infrastructure, to provisioning storage with snapshots, recovery and replication, your wildest sci-fi dream has come true. In his session at @DevOpsSummit at 20th Cloud Expo, Dan Florea, Director of Product Management at Tintri, will provide a ChatOps demo where you can talk to your storage and manage it from anywhere, through Slack and similar services ...
Mar. 24, 2017 06:30 PM EDT Reads: 4,111
SYS-CON Events announced today that SoftLayer, an IBM Company, has been named “Gold Sponsor” of SYS-CON's 18th Cloud Expo, which will take place on June 7-9, 2016, at the Javits Center in New York, New York. SoftLayer, an IBM Company, provides cloud infrastructure as a service from a growing number of data centers and network points of presence around the world. SoftLayer’s customers range from Web startups to global enterprises.
Mar. 24, 2017 05:15 PM EDT Reads: 1,295
SYS-CON Events announced today that Ocean9will exhibit at SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. Ocean9 provides cloud services for Backup, Disaster Recovery (DRaaS) and instant Innovation, and redefines enterprise infrastructure with its cloud native subscription offerings for mission critical SAP workloads.
Mar. 24, 2017 04:45 PM EDT Reads: 1,769
Have you ever noticed how some IT people seem to lead successful, rewarding, and satisfying lives and careers, while others struggle? IT author and speaker Don Crawley uncovered the five principles that successful IT people use to build satisfying lives and careers and he shares them in this fast-paced, thought-provoking webinar. You'll learn the importance of striking a balance with technical skills and people skills, challenge your pre-existing ideas about IT customer service, and gain new in...
Mar. 24, 2017 04:30 PM EDT Reads: 2,264
SYS-CON Events announced today that Juniper Networks (NYSE: JNPR), an industry leader in automated, scalable and secure networks, will exhibit at SYS-CON's 20th International Cloud Expo®, which will take place on June 6-8, 2017, at the Javits Center in New York City, NY. Juniper Networks challenges the status quo with products, solutions and services that transform the economics of networking. The company co-innovates with customers and partners to deliver automated, scalable and secure network...
Mar. 24, 2017 04:30 PM EDT Reads: 1,035
Interoute has announced the integration of its Global Cloud Infrastructure platform with Rancher Labs’ container management platform, Rancher. This approach enables enterprises to accelerate their digital transformation and infrastructure investments. Matthew Finnie, Interoute CTO commented “Enterprises developing and building apps in the cloud and those on a path to Digital Transformation need Digital ICT Infrastructure that allows them to build, test and deploy faster than ever before. The int...
Mar. 24, 2017 04:30 PM EDT Reads: 523
VeriStor Systems has announced that CRN has named VeriStor to its 2017 Managed Service Provider (MSP) 500 list in the Elite 150 category. This annual list recognizes North American solution providers with cutting-edge approaches to delivering managed services. Their offerings help companies navigate the complex and ever-changing landscape of IT, improve operational efficiencies, and maximize their return on IT investments. In today’s fast-paced business environments, MSPs play an important role...
Mar. 24, 2017 03:00 PM EDT Reads: 2,112