ENZYME REPLACEMENT THERAPY ON THE HORIZON....

Biomarin Annouced in June 2008 that they will be moving forward toward a Enzyme Replacement Therapy clinical trial for morquio.  www.morquiobmrn.com 

Biomarin is conducting a Morquio Clinical Assesment Study at two sites in the US. They hope to have 200 paricipants assessed by Dec 2009. This Morquio Clinical Assessment study is vital to gather info for the trial process and to include all ages and
severities. Biomarin is covering all the cost of the study and travel expenses
of participants and one caregiver. (that includes airfare, hotel, food, cab
fare to and from hospital!)

Listed below is contact info for both hospitals

Childrens Memorial Hospital-Chicago, IL
Rachel Katz (773) 880-3058 rkatz@childrensmemorial.org

Children's Hospital & Research Center Oakland California

Jo Ann Johnson (510) 428-3885 x5421   jajohnson@mail.cho.org

Biomarins Website for Morquio Trial: www.morquiobmrn.org  Register at this site to get regular updates about the trial.

Dr Tomatsu is also diligently working towards Morquio treatment.  Dr Tomatsu is a long time supporter and reseracher for Morquio  patients.  His dedication is strongly appreciated and we look forward to hearing more about his current research on 2nd generation enzyme replacement therapy for morquio A.  He is in the photograph below with other Morquio patients at a fundraiser in 2007.  Annabelle is the young one in his arms!

We are thrilled to see that Morquio ERT will most likely become a reality in the near future.  This development could make a world of difference to Annabelle's medical future.   As you might guess....This is FABULOUS news!!!