Dr. Maria Escolar at The University of North Carolina at Chapel Hill is conducting a natural history study of late infantile MLD.  The purpose of the study is to understand what happens to the neurodevelopment of children with MLD.  The information obtained from the study will help physicians understand if treatments like enzyme replacement infusions or cord blood transplantation are effective in improving or stabilizing the disease progression.  To see the invitation to participate in the study, please click here.  Click here to read the study details and criteria.  To learn more about Dr. Escolar and the Neurodevelopmental Function in Rare Disorders program at the University of North Carolina, click here.

On March 10, 2008, Zymenex, a Danish company, announced FDA approval of its Investigational New Drug (IND) application for its enzyme Metazym, which is intended for use in the treatment of MLD.  Phase 2 clinical trials will begin in the United States soon.  To read Zymenex's press release about the IND approval for Metazym, click here.  In April 2008, Zymenex was also granted Orphan Designation for Metazym, which qualifies the sponsor of the product for tax credits and marketing exclusivity incentives.  To read the press release about Metazym's Orphan Designation, click here.

As of April 24, 2008, Zymenex sold the rights to Metazym and its enzyme replacement therapy technology to Shire, plc.  Click here to read the full press release.  Shire is a US-based pharmaceutical company that has a history of working with enzyme replacement therapy and other drugs.  This experience, combined with its financial resources, will hopefully allow the ERT trials to progress.