MDA’s Statement on the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children voting to move the Duchenne muscular dystrophy nomination to full evidence review:

August 10th, 2023: Today, the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children (ACHNDC or “the Committee”) voted to move the nomination of Duchenne muscular dystrophy for the Recommended Uniform Screening Panel (RUSP) onto the next stage of review. The nomination now moves on to the “Evidence-based Review” stage (also known as “full evidence review”) in which the Committee examines the nomination further and ensures it is ready for recommendation to the RUSP.

MDA is grateful to the Committee for this positive outcome as we have urged the Committee to move the nomination to the next stage. 

Newborn screening is a public health program that screens nearly every newborn in the United States for certain conditions approximately 24 to 48 hours following birth. The conditions screened are serious conditions that if left untreated can have damaging impacts on the infant’s health. While newborn screening is a state-run program, the Federal government recommends a “uniform screening panel” to states in which each condition meets a set of criteria related to the viability of screening, the benefits and risks of screening each newborn for the condition, and the availability of follow-up treatment and care upon diagnosis. Already spinal muscular atrophy (SMA) and Pompe disease are included on the RUSP, and most states have both disorders included in their newborn screening panels.

Duchenne muscular dystrophy (DMD) was nominated in June 2022 by Parent Project Muscular Dystrophy (PPMD) with MDA as a co-sponsor of the package. Following months of initial review, a February 2023 vote against moving the nomination forward, and additional supporting evidence submitted earlier this Spring in support of the nomination, today’s vote by the ACHDNC is the culmination of well over a year of collaborative efforts across the Duchenne community to present the evidence necessary to move forward. We, with our partners at PPMD, will continue to work to build the supportive evidence and data necessary for an official Committee recommendation to the RUSP, hopefully sometime next year. 

MDA anticipates remaining active throughout the Committee’s continuing review, including testifying at quarterly meetings (just as we did this morning), and providing evidence and information to the Committee, when requested. We will continue to keep the neuromuscular disease community updated as the nomination process continues.