CURE MSD

Multiple Sulfatase Deficiency (MSD) is a genetic, rare and fatal condition that is extremely progressive, similar in effect to Alzheimer's or Parkinson's - but in children. Since United MSD Foundation's creation in 2016, advancements in gene therapy research have been made as we aggressively move closer to a cure... Community - We strive to create a sense of belonging, support, and empowerment by providing an environment for MSD families to build connections, alleviate isolation, amplify their voices, and share knowledge... Since inception, our mission has been to cure MSD. To that end, we have funded a gene therapy mouse model and subsequent toxicology study, and a Natural History Study. We run a MSD Biobank and Patient Registry to provide researchers with access to patient data and samples. Currently, we are supporting first-in-human gene therapy trials with our partners at the Children's Hospital of Philadelphia.

Primary location: Biloxi United States