In 2009, for example, University of California, Los Angeles, scientists discovered that children with a type of MPS disease called Sanfilippo syndrome produce high levels of tau, one of the two telltale proteins found in the brains of people with Alzheimer’s disease. The link means that better understanding the mechanism of Sanfilippo could lead to treatments for Alzheimer’s. At a recent lobbying day for rare disease advocates, Jill Wood, a mom from Brooklyn whose son has Sanfilippo type C, also known as MPS IIIC, tried to impress upon her local congressmen that although Sanfilippo research might directly affect only a few dozen children in the U.S., the greater good for society makes it a worthwhile investment.
by Lisa M. Jarvis |
May 13, 2013