MDA awarded a development grant (DG) totaling $170,873 over three years to Celine Baligand, a postdoctoral associate at the University of Florida College of Medicine in Gainesville. The funds will help support Baligand's research into the use of various imaging techniques to help determine the natural progression of Pompe disease (acid maltase deficiency or AMD). (MDA development grants are awarded to exceptional postdoctoral candidates who have the best chance of becoming independent researchers and future leaders of neuromuscular disease research.)
The current therapeutic approach for Pompe disease, enzyme replacement therapy or ERT, has improved outcomes for people with the disease. Alternatives to ERT, such as gene therapy, are under development.
Baligand and colleagues plan to develop tools, including magnetic resonance (MR) spectroscopy and other imaging techniques, for use in the noninvasive study of the natural course of Pompe disease in a mouse model. Such tools will facilitate the development of second- and third-generation treatment strategies for the disease.
"The development of MRI technology is crucial to improving the treatment of muscle diseases," Baligand said, noting that the development and validation of appropriate procedures for MRI could inform the preclinical testing of drugs and gene therapy in Pompe mouse models, potentially speeding the development of treatments for people with the disease.
Funding for this MDA grant began February 1, 2012.