PTC Therapeutics and Parent Project Muscular Dystrophy Advance Drug Discovery Program to Improve Heart Function in Duchenne/Becker Muscular Dystrophy Patients
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.
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