Clinical trial: Lumizyme

Avery is no longer going to be on Myozyme and will be switching to Lumizyme. Why? The way WE understand it...there was a manufacturing issue with Myozyme that created a shortage as well as Myozyme is made in smaller batches...so kids are using more Myozyme because they are getting bigger and living longer so it is getting used up faster. The dosage is based on weight so as kids get bigger they use more. Where does Lumizyme come into the picture? Lumizyme is used in Europe in Pompe patients currently. It is basically the same med just manufactured differently in bigger quantities. Genzyme(the company that makes both) is trying to get and FDA indication for Lumizyme in the USA for infantile Pompe. Since Avery in not involved in a study with Myozyme we really had no other choice but to do this trial.

Avery playing Angry Birds while waiting for tests

Getting her EKG and eating a sucker

With Debbie playing Angry Birds and getting ready to get vitals done

To get ready for this trial we had to make several trips to Children's in Detroit to sign consent forms and do baseline testing. One of the first trips we made was to sign consent forms. There were two basic consent forms and one autopsy consent form. The autopsy consent form kinda freaked me out. It was giving consent to perform an autopsy if something did happen to Avery. **Gulp**If that doesn't put things into perspective I don't know what does. At another time we went to Children's for a PT evaluation, an echo cardiogram and and EKG. Avery did so great during all these tests. She held perfectly still for the heart tests and didn't shed a tear...for a 2 year old I think that is pretty great. Next up...the first Lumizyme infusion on 4/27/12. Wade is going with me for this one.