Genzyme Update on the LOTS Trial- January 2007

In an effort to complete the AGLU02704 study (Late-Onset Treatment Study, LOTS) as responsibly as possible, Genzyme has amended the study protocol to incorporate the use of an adaptive design. Genzyme utilized this method to help ensure a conclusive trial outcome in...

SARI

Background We are the parents of Sari, a Belgian girl of 4.5 years old suffering from infantile Pompe´s disease.  Based on the normal development of the disease, Sari would have passed away already, probably before her 1st birthday. However she has been receiving an...