The National MPS Society has funded more than $4 million toward research. These research dollars have accomplished much over the years. In April 2003, the first enzyme replacement therapy( ERT) for an MPS disease was approved by the FDA, Aldurazyme for MPS I. Naglazyme, the ERT for MPS VI was approved in 2005, and in 2006 the FDA approved Elaprase, ERT for MPS II. Getting the enzyme into the brain is the next step, and clinical trials are in progress in MPS I and MPS II with protocols being written for these studies in MPS II and MPS III. Children affected with ML, MPS3, MPS4, and MPS7 are still without treatment. Although great strides have been made in research, continued funding is urgently needed to accelerate the encouraging research that is bringing therapies and ultimately the cures — to children and adults with MPS and related diseases.
The Society impacts its members through family support programs, research for treatments and cures, voices in Washington, DC, hope through conferences and support for bereaved families. Families are the recipients of hope and support throughout their journey with MPS. These are rare diseases, and families have few places to turn for continued support year after year. The Society holds conferences every year in different parts of the country, enabling families to meet and learn more about their disease. Leadership families have been identified both by region and by syndrome to serve as valuable points of contact for newly diagnosed families. In addition, a fund has been designated to assist families to attend Society conferences, assist with trips to medical appointments, to obtain medical products that will improve their quality of life and to provide continuing education scholarships. The Society also provides much needed emotional support to all those affected by the tragedies of MPS. Your funds are critical in order to assist families with information they need to provide the best care for their children.
When my son was diagnosed with MPS II, in July 2011, the MPS Society, through its family support program, provided me immediate access to the National Conference. This gave me priceless information on treatments needed for my son that the doctors at my local, and well respected children hospital, could not provide me. Without the Society, I would not have known about critical treatments that my son needed. These ended up being essential to his quality of life. For that, I am forever grateful.
Lisa Todd, MPS II parent