September 18, 2009

We came back to Univ of Minnesota Children's Hospital to Unit 5D to find our spunky, active, crazy, back to normal baby! She's doing great! Right now we are waiting for her 1st dose of IV enzyme. Laronidase (Aldurazyme{TM}) belongs to a class of drugs called enzyme replacement therapies (ERT) that provides people with sufficient quantities of an important enzyme that they cannot create on their own. The main ingrediant in laronidase is a protein that is identical to a naturally occurring form of the human ezyme alpha-L-iduronidase. Laronidase replaces the missing enzyme and and restores sufficient enzyme activity to break down glycosaminoglycan (GAG) build-up. In a clinical study, laronidase ERT decreased abnormally high GAG substance in urine and decreased liver size, improved lung problems and corrected symptoms such as sleep apnea and airway problems. However, laronidase ERT alone alone has not shown to benefit the progressive neurocognitive decline or improve survival. This is the reason that Dr. Orchard and the University of Minnesota are doing research and clinical trials giving laronidase Intrethecally (IT). Laronidase given intraveneously (IV) does not cross the blood-brain barrier. By giving the enzyme IV and IT, neurocognitive abilty is being being preserved.

Wow. That was lengthy! But you know how windy I am! So IV ERT is in progress while I type. It feels like every drop that goes in is buying our girly just one more day with us.