Click here to close now.


News Feed Item

MyoKardia Announces Launch of SHaRe Patient Registry for Genetic Heart Disease in Collaboration with World-Leading Cardiovascular Patient Care Centers

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.

About MyoKardia

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

More Stories By Business Wire

Copyright © 2009 Business Wire. All rights reserved. Republication or redistribution of Business Wire content is expressly prohibited without the prior written consent of Business Wire. Business Wire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
In recent years, at least 40% of companies using cloud applications have experienced data loss. One of the best prevention against cloud data loss is backing up your cloud data. In his General Session at 17th Cloud Expo, Sam McIntyre, Partner Enablement Specialist at eFolder, presented how organizations can use eFolder Cloudfinder to automate backups of cloud application data. He also demonstrated how easy it is to search and restore cloud application data using Cloudfinder.
With major technology companies and startups seriously embracing IoT strategies, now is the perfect time to attend @ThingsExpo 2016 in New York and Silicon Valley. Learn what is going on, contribute to the discussions, and ensure that your enterprise is as "IoT-Ready" as it can be! Internet of @ThingsExpo, taking place Nov 3-5, 2015, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with 17th Cloud Expo and will feature technical sessions from a rock star conference faculty ...
Internet of @ThingsExpo, taking place June 7-9, 2016 at Javits Center, New York City and Nov 1-3, 2016, at the Santa Clara Convention Center in Santa Clara, CA, is co-located with the 18th International @CloudExpo and will feature technical sessions from a rock star conference faculty and the leading industry players in the world and ThingsExpo New York Call for Papers is now open.
The cloud. Like a comic book superhero, there seems to be no problem it can’t fix or cost it can’t slash. Yet making the transition is not always easy and production environments are still largely on premise. Taking some practical and sensible steps to reduce risk can also help provide a basis for a successful cloud transition. A plethora of surveys from the likes of IDG and Gartner show that more than 70 percent of enterprises have deployed at least one or more cloud application or workload. Y...
Cloud computing delivers on-demand resources that provide businesses with flexibility and cost-savings. The challenge in moving workloads to the cloud has been the cost and complexity of ensuring the initial and ongoing security and regulatory (PCI, HIPAA, FFIEC) compliance across private and public clouds. Manual security compliance is slow, prone to human error, and represents over 50% of the cost of managing cloud applications. Determining how to automate cloud security compliance is critical...
Growth hacking is common for startups to make unheard-of progress in building their business. Career Hacks can help Geek Girls and those who support them (yes, that's you too, Dad!) to excel in this typically male-dominated world. Get ready to learn the facts: Is there a bias against women in the tech / developer communities? Why are women 50% of the workforce, but hold only 24% of the STEM or IT positions? Some beginnings of what to do about it! In her Day 2 Keynote at 17th Cloud Expo, San...
As organizations shift towards IT-as-a-service models, the need for managing & protecting data residing across physical, virtual, and now cloud environments grows with it. CommVault can ensure protection & E-Discovery of your data - whether in a private cloud, a Service Provider delivered public cloud, or a hybrid cloud environment – across the heterogeneous enterprise.
In his keynote at @ThingsExpo, Chris Matthieu, Director of IoT Engineering at Citrix and co-founder and CTO of Octoblu, focused on building an IoT platform and company. He provided a behind-the-scenes look at Octoblu’s platform, business, and pivots along the way (including the Citrix acquisition of Octoblu).
In his General Session at DevOps Summit, Asaf Yigal, Co-Founder & VP of Product at, explored the value of Kibana 4 for log analysis and provided a hands-on tutorial on how to set up Kibana 4 and get the most out of Apache log files. He examined three use cases: IT operations, business intelligence, and security and compliance. Asaf Yigal is co-founder and VP of Product at log analytics software company In the past, he was co-founder of social-trading platform Currensee, which...
Today air travel is a minefield of delays, hassles and customer disappointment. Airlines struggle to revitalize the experience. GE and M2Mi will demonstrate practical examples of how IoT solutions are helping airlines bring back personalization, reduce trip time and improve reliability. In their session at @ThingsExpo, Shyam Varan Nath, Principal Architect with GE, and Dr. Sarah Cooper, M2Mi’s VP Business Development and Engineering, explored the IoT cloud-based platform technologies driving t...
There are over 120 breakout sessions in all, with Keynotes, General Sessions, and Power Panels adding to three days of incredibly rich presentations and content. Join @ThingsExpo conference chair Roger Strukhoff (@IoT2040), June 7-9, 2016 in New York City, for three days of intense 'Internet of Things' discussion and focus, including Big Data's indespensable role in IoT, Smart Grids and Industrial Internet of Things, Wearables and Consumer IoT, as well as (new) IoT's use in Vertical Markets.
The Internet of Things (IoT) is growing rapidly by extending current technologies, products and networks. By 2020, Cisco estimates there will be 50 billion connected devices. Gartner has forecast revenues of over $300 billion, just to IoT suppliers. Now is the time to figure out how you’ll make money – not just create innovative products. With hundreds of new products and companies jumping into the IoT fray every month, there’s no shortage of innovation. Despite this, McKinsey/VisionMobile data...
We all know that data growth is exploding and storage budgets are shrinking. Instead of showing you charts on about how much data there is, in his General Session at 17th Cloud Expo, Scott Cleland, Senior Director of Product Marketing at HGST, showed how to capture all of your data in one place. After you have your data under control, you can then analyze it in one place, saving time and resources.
SYS-CON Events announced today that Alert Logic, Inc., the leading provider of Security-as-a-Service solutions for the cloud, will exhibit at SYS-CON's 18th International Cloud Expo®, which will take place on June 7-9, 2016, at the Javits Center in New York City, NY. Alert Logic, Inc., provides Security-as-a-Service for on-premises, cloud, and hybrid infrastructures, delivering deep security insight and continuous protection for customers at a lower cost than traditional security solutions. Ful...
Just over a week ago I received a long and loud sustained applause for a presentation I delivered at this year’s Cloud Expo in Santa Clara. I was extremely pleased with the turnout and had some very good conversations with many of the attendees. Over the next few days I had many more meaningful conversations and was not only happy with the results but also learned a few new things. Here is everything I learned in those three days distilled into three short points.