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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. Becerra stated:
“After considering the utility of current screening technologies, treatment for MPS II, and the impact on public health systems, I accept the Committee’s recommendation to expand the Recommended Uniform Screening Panel to include the addition of MPS II.”
Conditions listed on the RUSP are provided to individual states as a recommendation for adoption for newborn screening.
This nomination, led by National MPS Society President/CEO Terri Klein and Chief Scientific Officer Matthew Ellinwood, was the product of years of work and collaboration, publications, and a community with a purpose. Klein shares:
“This is a momentous occasion for the MPS II Patient Community. Today, we can begin identifying newborns with MPS II, Hunter Syndrome. I am grateful to DHHS for expanding the RUSP and recognize this will improve the lives of newborns and infants across our country.
Screening newborns for MPS II will provide equitable access to immediate, life-saving therapies. Equitable access will provide testing to everyone, including traditionally under-served communities. Since enzyme replacement therapy (ERT) has been available for our boys, we have witnessed increased quality and length of life and an opportunity for young men to thrive in the world.
Now, early access will erase many of the debilitating manifestations of this disease. Newborns treated with ERT will have access to eventual therapies that address cognitive decline that are being developed and in clinical trials today.”
We would like to extend thanks to the ACHDNC for their newborn screening review. We also recognize and appreciate the families and individuals with MPS II who shared their stories and experiences and the thousands of signatories who contributed to the letter of support provided to the ACHDNC.
MPS II is the second MPS disorder to be included on the RUSP. MPS I was added by DHHS in 2016. Today, 30 states screen for MPS I. Our efforts to add newborn screening for all MPS diseases remain at the forefront of our work. With MPS I and MPS II now on the RUSP, the Society will submit a request for MPS VII, Sly Syndrome, in the coming months. We are investigating paths forward to present requests for MPS III, IVA, and VI and continue to prioritize this work, recognizing this as an effort that supports preservation of physical and cognitive functioning for babies diagnosed and directly saves lives.
To access the letter from the Department of Health and Human Services, please click here.