Research We Support
Background Info
As a rare “orphan” disease, funding for MPS1 research faces an uphill battle. It is important to understand that because of advances in diagnosis and treatment, individuals with MPS1 now have a chance for a longer life. Because of these successes, researchers now have an opportunity to confront the many complications of the disease and work on discovering new treatments for improving the quality of life for people with MPS1. Since MPS1 is one of many types of lysosomal storage diseases, new research into MPS1 may also lead to advancements in other related diseases.
Supported Research
Our goal is to support promising research into new treatments for those suffering from MPS1. Within six months of our founding, we were able to fund our first research project with a $32,500 grant to The Intra-articular Enzyme Replacement Therapy on a Canine Model for MPS1 Study, led by Dr. Raymond Wang and Dr. Afshin Aminian of CHOC Children’s Hospital in Orange, CA. This will be an important first step in studying the potential of using direct injections of enzyme replacement therapy (ERT) into the joints to treat the severe pain and inflammation caused by MPS1. This study will hopefully provide a better understanding, and a potential new treatment, for joint pain and stiffness that people with MPS1 suffer with every day.
As this study moves forward, we are continuing to meet with other researchers such as Dr. Simonaro whose work also shows promise for uncovering potential new treatments to ease the pain and suffering endured every day by those with MPS1. The key mission of the MPS1 Research Foundation is to find more effective treatments for individuals suffering from MPS1. We are aggressively seeking out new and cutting-edge research by pursuing the best and the brightest scientists currently working in the field of lysosomal storage diseases.
Following are brief outlines of the work currently being conducted by leading researchers that funds raised by the MPS1 Research Foundation will help support. We will update the latest status of these important research projects here on the Website, through our email newsletter, and by using Facebook. We encourage you to keep in touch with us as we work hard to raise money in support of this important work.
Dr. Calogera M. Simonaro, PhD
Department of Genetics and Genomic Sciences
Mt. Sinai School of MedicineNew York, NY
Dr. Simonaro is conducting very important research into one of the most challenging problems facing people with various forms of MPS – the pain and disfigurement to their bones and joints as the disease progresses. Despite the success of enzyme replacement therapy and bone marrow transplants, there continues to be a painful and debilitating degeneration in movement and flexibility for people suffering from MPS. Dr. Simonaro is hopeful that her work will lead to “fast-track” approval for FDA-approved anti-inflammatory drugs such as Remicade™ to help improve the lives of MPS patients.
Learn More About Research by Dr. Simonaro
Dr. Raymond Wang, MD
Division of Metabolic Disorders
Children’s Hospital of Orange County (CHOC)
Orange, CA
Dr. Wang is board-certified in pediatrics, clinical genetics, and biochemical genetics. He is studying novel ways to treat and measure treatment efficacy in patients with various different storage disorders. Dr. Wang established a multidisciplinary lysosomal storage disorder (LSD) clinic at CHOC where LSD patients, including those with MPS1, can be seen by several sub specialists at the same clinic visit. He has proposed an important new study designed to further advance previous studies to examine the potential of intra-articular delivery of enzyme into major joint spaces to directly treat the pain and lack of mobility faced by MPS1 individuals as their disease progresses.
Learn More About Research by Dr. Wang
