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MyoKardia Announces Launch of SHaRe Patient Registry for Genetic Heart Disease in Collaboration with World-Leading Cardiovascular Patient Care Centers
|By Business Wire
|May 14, 2014 08:09 AM EDT
Inc., a company pioneering the development of precision therapies
for genetic heart disease, today announced the launch of the Sarcomeric
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,
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.
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