MTAP Fact Sheet July 2007
MTAP FACT SHEET JUNE 2007
Myozyme® Temporary Access Program
Pompe Program Update May 2007 There are two studies to further evaluate Myozyme® (alglucosidase alfa) that will be conducted as part of Genzyme’s postmarketing commitments (PMC) to the FDA. A postmarketing commitment is an agreement between a regulatory agency and a...
Flying with a ventilator in the USA
GENZYME POMPE PROGRAM APRIL 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...
Expensive drug gives hope to rare disease sufferer
BioMarin is currently seeking patients for a Phase 3 clinical trial in Late Onset Pompe Disease. This phase 3 study incorporates a switchover design in which all patients switch directly from Myozyme® or Lumizyme®* to the experimental drug BMN 701 without missing...
Amicus Therapeutics Announces First Patient in Phase 2 Study for Pompe Disease