May 15 is International MPS Awareness Day.

Before I recall my own thoughts on MPS Awareness Day, I wanted to let you know what this day is for:
On International MPS Awareness Day we:

• Remember all the children and adults who suffer from MPS and related diseases.
• Think about the children we have lost.
• Think about the doctors and scientists who are dedicated to finding a cure for MPS and related diseases.
• Remember each other and be thankful for the strength and support we both give and receive.

In honor of International MPS Awareness Day, the International MPS Network launched a website http://www.impsn.org/ honoring everyone globally who has MPS and their families. The National MPS Society is asking everyone to send ecards and publicize this day.

What I think about on a day like International MPS Awareness Day, are the kids we couldn’t save with MPS I during the first study. We had so much hope but in many cases the disease was too far along and the treatment not able to reverse some of the most important problems in the heart and the skeleton. I am writing a book now of my experiences developing Aldurazyme for MPS I and the 10 families that participated. We still have four of the ten kids with us now 12 years later. Ryan has had 12 years of enzyme therapy and recently had his 22^nd birthday.

Among the ones we lost, I remember the excitement when everyone was getting better but then we could not fix everything. Eventually something happened, a surgery or an illness, and then we lost patients, one by one. These great kids and families did have a chance to feel hope for once and feel better, but I hoped we could give them more. It also pains me to know that if we had treated them at a younger age before the disease had progressed, we might have saved them. Once their hearts were damaged, or the bones bent, it was hard to prevent the steps eventually leading to their loss.

As we remember them, we have to recognize that other MPS and ML diseases are in this situation now, potentially treatable but not yet treated. The longer we wait many of these kids will become too far along to be helped. Transforming the regulatory process to help encourage more investment and development for MPS and ML diseases is now our important goal. I am happy for what we have accomplished in treating MPS diseases but I cannot forget those we lost and those we have not yet treated.