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BioMarin Pharmaceutical is currently conducting two clinical studies for MPS IVA (Morquio syndrome): MorCAP (a clinical assessment program that started in October 2008) and a Phase 1/2 clinical study (initiated in April 2009). Individuals who have participated in the International Morquio Organization (IMO) survey are welcome and encouraged to participate in MorCAP.

MorCAP (Morquio A Clinical Assessment Program)

MorCAP [Study MOR-001] has been designed to provide a fuller understanding of MPS IVA syndrome by measuring various aspects of the disorder including endurance and respiratory function in affected patients. These insights may help BioMarin design future clinical studies. Participation in MorCAP will require one or more visits to a clinic or hospital, but no experimental drug is administered during theses visits. MorCAP is a multinational study and will likely include sites located in the US, UK, South America, Canada, Europe, and Asia.

Phase 1/2 Study

The Phase 1/2 study [Study MOR-002] was initiated in the United Kingdom in April 2009. This study includes weekly administration of an experimental enzyme replacement therapy. Enrollment in this study is limited. The goal of MOR-002 is to assess safety and to choose the best dosing regimen for MPS IVA patients. The Phase 1/2 endpoints will include changes in plasma and urine keratan sulfate, respiratory function, and measures of endurance (such as a walk test). The results of this study will be analyzed to help determine the possible next steps in developing this potential therapy.

To learn more about participating in MorCAP or the Phase 1/2 study, please speak to your physician, or register here to receive updates on the latest developments.