The SSADH Association awarded $55,000 to Henry Lee, Ph.D

The SSADH Association awarded $55,000 to Henry Lee, Ph.D., Alexander Rotenberg, Ph.D., MD, and Phil Pearl, MD from Boston Children’s Hospital / Harvard Medical School to continue evaluating the potential for Enzyme Replacement Therapy for SSADH patients.

The grant period will begin July 1st and run for six months to evaluate the optimal enzyme restoration parameters in the SSADH inducible mouse model.  The goal of this grant is to evaluate three critical aims for enzyme replacement therapy (rate, age and location) using the mouse model.  

We are hopeful that the additional six months with help to position this research for further funding by federal and/or private sectors. This project is an extension from the initial project to create the inducible mouse model.