SOUTH PLAINFIELD, N.J. and HACKENSACK, N.J., Feb. 1, 2011
/PRNewswire/ — PTC Therapeutics, Inc. (PTC) and Parent Project
Muscular Dystrophy (PPMD) today announced an expansion of their
collaboration to discover new treatments for patients with
Duchenne/Becker muscular dystrophy (DBMD). PPMD has awarded
PTC a $250,000 grant, with the potential of additional funding, to
advance drug discovery efforts in identifying a new treatment that
improves heart function in patients with DBMD.
(Logo: http://photos.prnewswire.com/prnh/20010919/PTCLOGO)
PPMD’s President Pat Furlong explained the decision for the
grant, “The fight to end Duchenne and Becker muscular dystrophies
must involve a comprehensive approach, considering all of the
critical muscles that make up the human body. The heart is a
muscle too, and Parent Project Muscular Dystrophy has made cardiac
health a top priority within our research investment for 2011.
PTC Therapeutics understood the importance of cardiac health
from the moment we agreed to collaborate with them, now over ten
years ago. PTC continues to make our community’s priorities
their own and we are hopeful their approach to increase the level
of SERCA2a protein will translate into healthier hearts for our
sons.”
Heart failure is a leading cause of death in patients with
advanced DBMD. Through the course of the disease, heart
function is progressively compromised by calcium overload and
inadequate transport, which leads to cell imbalance, dysfunction
and death. SERCA2a is a calcium ion pump found in heart
tissue. The goal of this research program is to identify new
medicines that increase the amount of SERCA2a in the heart,
resulting in improvements in calcium ion transport and heart
function. Using its proprietary drug discovery technology,
PTC has identified early stage compounds that increase levels of
SERCA2a prot
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