Yesterday, we received news that the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) has been terminated, effective immediately.

For over 20 years, this committee has served as the guiding body for adding conditions to the Recommended Uniform Screening Panel (RUSP)—a critical step in ensuring that newborns across the country have access to early diagnosis and intervention for serious and/or life threatening childhood disorders. The immediate dissolution of the ACHDNC raises significant concern for the newborn screening community and the rare disease community at large.

For over a decade, PPMD has been at the forefront of advancing newborn screening for Duchenne—designing, funding, and collaborating on a years-long pilot program in New York State that succeeded despite taking place during the height of the COVID-19 pandemic; leading the nomination package to add Duchenne to RUSP; and driving policy change through meaningful state-based advocacy. 

This decision leaves families, advocates, and experts questioning what comes next for the future of newborn screening in the United States.

Despite this uncertainty, PPMD remains committed to our mission. We are actively collaborating with leading newborn screening experts to determine the best path forward for our nomination package. Our goal remains unchanged: to ensure that every baby born in the U.S. has the opportunity for early detection of Duchenne, leading to earlier care, better outcomes, and the chance for timely, life-changing treatments.

This setback will not stop us. Over the coming weeks, we will work with our partners, scientific experts, and policy leaders to explore alternative pathways for advancing Duchenne newborn screening. We will continue to fight for families who deserve attainable answers, support, and access to care from the very start of their child’s life.

PPMD has always been driven by the belief that no challenge is insurmountable. While the dissolution of the ACHDNC presents a significant hurdle in our newborn screening strategy, we will not waver in our commitment to the Duchenne community. We urge our community to stay engaged, continue advocating, and stand with us as we navigate this evolving landscape.

To the dedicated public servants of the ACHDNC—your tireless work, thoughtful challenges, and unwavering commitment have shaped the very foundation of newborn screening in this country, and without you, we would not be where we are today. We are profoundly grateful and hopeful that you will continue alongside us on this journey in new and meaningful ways.

We will keep each of you informed as we learn more and develop our next actions – for which we undoubtedly need your advocacy. In the meantime, thank you for your continued support and determination. Together, we will push forward and find a way to ensure that Duchenne is included in newborn screening nationwide.