ENZYME REPLACEMENT THERAPY ON THE HORIZON....

In the past two years, the Morquio community has seen real research progress for treatment.   In 2008, our Mad Hatter Fundraiser raised approx $34,000.  National MPS Society matched our funds to create a Morquio research grant of $80,000 which was rewarded to Calogero Simonaro, Ph.D.  Her goal of research is to develop and evaluate new treatment approaches for two important organ systems in Mucopolysacharisoses, the bones and joints.   Our fundraiser provided the opportunity for this research!!!

Another gigantic lead has been with BioMarin.  BioMarin is conducting clinical trials for Morquio Syndrome Enzyme Replacement Therapy (ERT).  The phase I/II of the study were completed in the UK with success.  Phase III is to begin in the USA and UK in 2011.  ERT will replace some of the enzyme that Annabelle is missing genetically and theoretically slow the progression of her disease.  This is a momentous leap in research for Morquio Syndrome toward an actual treatment.  Go to www.bmrn.com for info about trial     

Annabelle is highlighted on BioMarins website: http://www.bmrn.com/patients-physicians/patient-stories/annabelle.php   (so cool!!!!)

Biomarin is also conducting a Longitudinal Morquio Clinical Assesment Study at two sites in the US. They hope to have 200 paricipants. 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!)  www.morquiobmrn.com

If you live near or in the DC Metro area, please contact me.  I would love to develop a network of Morquio patients and families. 703-256-1980

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

Dr Tomatsu and Dr Adriana Montana are also diligently working towards Morquio treatment.  Dr Tomatsu and Dr Adriana Montana are a long time supporters and researchers for Morquio  patients.  Their dedication is strongly appreciated and we look forward to hearing more about their current research on 2nd generation enzyme replacement therapy, gene therapy, and substrate reduction therapy for Morquio A.  Below is in the photograph of Dr Tomatsu with other Morquio patients at a fundraiser in 2007.  Annabelle is the young one in his arms!