Curemsd

Our mission is simple, yet powerful: To cure Multiple Sulfatase Deficiency. Multiple Sulfatase Deficiency (MSD) ia a rare genetic, progressive, neurodegenerative disease caused by the build up of cellular waste throughout the body leading to neurologic regression and multisystemic symptoms. Children with MSD rarely survive past their 13th birthday. Currently there is no treatment or cure available for MSD; however, there is hope. Various mutations of the SUMF1 gene are known to be the cause of MSD, and the knowledge of these specific gene mutations allows researchers and doctors to move forward and find a treatment and cure. Gene therapy could help slow the progression of the disease, and possibly deliver a cure. The United MSD Foundation is currently working with partner organizations, researchers, and doctors from all over the world to fund a first-in-human gene therapy clinical trial. Additional initiatives include continuing to identify patients worldwide, the development of a patient registry and biobank, and advocating for newborn screening across the United States.