Join Elsa Shapiro, PhD and Paul Harmatz, MD for an informative conversation on the unique role of the consulting psychologist, building partnerships within the greater trial ecosystem, and considerations for protocol development.
The annual award recognizes exceptional patient leadership advocacy in the field of lysosomal disease. Presentation will take place Monday, February 8, 2021 at the 17th Annual WORLDSymposium.
The recommended framework would qualify many individuals with MPS and ML to be among the first recipients of a COVID-19 vaccine when it becomes available.
National MPS Society Bake Sale and Bake Off starts October 30 - November 7!
Our Virtual Family Conference has concluded but registered attendees can still access it on-demand through October 12th!
Now recruiting patients born in: California, Michigan, New Jersey, New York This study is also recruiting any patients in any state who have privately banked cord blood and would provide a single-drop sample. Please contact Leslie Urdaneta directly if interested. This study is recruiting patients with MPS I (attenuated: Hurler-Scheie or Scheie Syndromes), MPS II, … Continued
Gene Therapy Updates and Pharmacological PresentationsMatthew Ellinwood, DVM, PhDCSO, National MPS Society Dr. Matthew Ellinwood spoke on advances in a subset of gene therapy that involves gene editing. Gene editing involves the targeted and sequence specific editing of genomic DNA in a patient or a patient’s cells. Various methods exist involving systems developed from discoveries … Continued
National MPS Society AdvocacyChristine Tippett, Chair, Advocacy CommitteeNational MPS Society Board of Directors The National MPS Society’s Advocacy Committee has monthly calls. There are over 25 members and they work in areas of newborn screening, appropriations language, legislative action alerts to members, COVID task force and partner with Everylife Foundation and the RDLA. In 2020, … Continued
Accelerating a Treatment for GNTAB-Related Disorders Richard Steet, Ph.D.Jennifer Klein, MS One year since the formation of the MCRN, the network continues to meet monthly, with the primary function to coordinate several labs focused on ML research. The Network executes systematic and standardized testing of different treatment ideas in common animal models for ML II, ML … Continued
The Impact of COVID-19 on MPSHeather Lau, MD The COVID-19 global pandemic has made 2020 a year like no other. In her keynote presentation, Dr. Heather Lau delivers an overview of the disease, some recent U.S. statistics, and the varying ways it can impact the MPS community. The most common symptoms of COVID-19 have been … Continued