The Muscular Dystrophy Association has given a $300,000 grant to the Center for Genetic Medicine Research (CGMR) at Children’s National Medical Center in Washington, D.C., to continue standardized and reliable preclinical studies of neuromuscular diseases.
The MDA Translational Research Grant was awarded to CGMR's Kanneboyina Nagaraju, who directs the Murine Functional Testing Core Facility. It's the largest preclinical drug testing facility for neuromuscular disease models.
The facililty helps advance drug development by testing drugs and other experimental treatments in mouse models of neuromuscular disease, allowing researchers to obtain the best possible information about how a treatment is likely to influence a disease course.
The U.S. Food and Drug Administration (FDA) generally requires this type of preclinical study before it allows new substances to be tested in humans.
Several strains of mice with various neuromuscular diseases are maintained at the facility, including some with rare forms of muscular dystrophy that are not widely available.
Using state-of-the-art equipment and techniques for its mouse testing, the facility performs:
The facility was previously funded by the U.S. Department of Defense, but this funding is no longer available.
"MDA is proud to step in to make sure this valuable facility continues operating," said Jane Larkindale, director of MDA’s translational research program. She noted that many MDA-associated researchers and companies consider the facility a vital service.
The grant was made through MDA's translational research program, which is designed to bridge the gap between laboratory research and drug development for the neuromuscular diseases under the Association’s umbrella.
Nagaraju, who holds doctoral degrees in veterinary medicine and immunology, is a principal investigator at CNMC who has previously received MDA research support and is a member of MDA's Medical Advisory Committee.