DHHS adds MPS II to the RUSP for Newborn Screening in the United States

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.

Financial help available for MPS VII patients

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

FDA approves MPS VII drug

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 treats first patient with gene therapy

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

Society awards MPS and ML research grants

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