International Pompe Association

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As some of you are probably aware, Genzyme has announced that, due to production and supply constraints, starting March 30, 2012 the 160 liter Myozyme (which is ONLY used in the United States) will no longer be shipped to patients over the age of 12 months.

This ONLY affects patients in the United States, and it ONLY affects those who are currently receiving the 160 liter Myozyme product who are over 12 months old. (If you are currently receiving treatment outside the United States or are being treated with Lumizyme inside the United States, you are receiving product from the 4000 liter bioreactors and you will NOT be affected.)

In order to ensure uninterrupted access to therapy for patients over the age of 12 months who are currently on the 160 liter Myozyme (about 100 patients), Genzyme has enacted the ADVANCE study. Through this study patients will recieve enzyme produced at the 4000 liter scale, which is called Lumizyme in the United States and Myozyme everywhere else in the world.

I know that this news raises a lot of questions. Please take a moment and visit the AMDA (www.amda-pompe.org) or IPA (www.worldpompe.org) websites where you will find:

  1. a Pompe Program Update from Genzyme explaining the situation and how you may or may not be affected;
  2. the ADVANCE Fact Sheet, which will answer some questions about the study; and
  3. Statements from the AMDA and IPA concerning this situation.

In the coming days and weeks one thing to remember is that the 4000 liter product (Lumizyme in the US and Myozyme everywhere else) is being used everywhere else in the world with good results. I have spoken with Dr. Priya Kishnani, the leading Pompe Disease expert in the United States, and she has asked me to pass on the following: As many of you know, I have had the privilege of working with infants all over the world who are affected by Pompe disease and in my experience these babies are doing well on the 4000L product; additionally, data from the Taiwan newborn screening studies has been reassuring with regard to infants on Lumizyme.

With that said, if you have any questions or concerns please do not hesitate to contact me (TiffanyLHouse@aol.com) or the IPA and I will do our best to address them and get you the answers you need. I have been working closely with Genzyme, the IPA, and Dr. Kishnani and will continue to do so to bring you the most up-to-date information possible regarding this situation.

Tiffany House
(AMDA President and IPA Vice-Chair)

 

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