August 3, 2022 - The National MPS Society announces that Xavier Becerra, the Secretary of the Department of Health and Human Services (DHHS) has approved adding MPS II as a condition to the recommended uniform screening panel (RUSP) for newborns. This long-awaited acceptance follows the Advisory Committee on Heritable Disorders in Newborns and Children’s (ACHDNC) approval to move the nomination forward to DHHS after a considerable evidence review.
The combination of efficacy and safety data on intravenous AAV9 gene therapy from animal models and the preliminary data from human trials are highly encouraging. We therefore believe that intravenous … Continued
Company will review data; analyze positive trends beyond initial results Shire announced today that top-line data from its phase II/III clinical trial for MPS II did not meet primary or … Continued
The Assistance Fund, an independent charitable patient assistance foundation that helps patients and families facing high medical out-of-pocket costs, has launched a financial support initiative for MPS VII patients and … Continued
The U.S. Food and Drug Administration today approved Mepsevii (vestronidase alfa-vjbk) to treat pediatric and adult patients with MPS VII, Sly syndrome. The safety and efficacy of Mepsevii, developed by … Continued
Sangamo Therapeutics treated its first patient in the Phase 1/2 clinical trial (“the CHAMPIONS study“) evaluating SB-913, an investigational in vivo genome editing therapy for people with MPS II, Hunter … Continued
The National MPS Society’s annual research program has awarded $405,000 in funding for additional research grants. Dr. Sharon Byers – $90,000 general grant over two years SA Pathology (WHC site) … Continued