Secretary Kennedy Adds Duchenne Muscular Dystrophy, Metachromatic Leukodystrophy to Newborn Screenings

WASHINGTON—DECEMBER 16, 2025 — The U.S. Department of Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr. today approved the addition of Duchenne Muscular Dystrophy (DMD) and Metachromatic Leukodystrophy (MLD) to the Recommended Uniform Screening Panel (RUSP) following scientific review and public comment. Early detection of both conditions allows children to receive FDA-approved therapies at the most effective time, helping to slow disease progression and preserve their quality of life.

“As my uncle has said, ‘although children may be the victims of fate, they will not be the victims of our neglect,’” said Secretary Kennedy. “This action honors that principle. Early screening gives every child a fair chance at timely, effective care, and it delivers families the answers and treatment options they deserve—right when they need them most.”

Most children with DMD or MLD are diagnosed at four to five years of age, when significant muscle loss or functional decline has already occurred. Not only will adding these conditions to the RUSP help children retain abilities for a longer period, but it also gives families a better chance to avoid the long delays, repeated specialist visits, and financial and emotional strain that often define the years-long diagnostic search for rare diseases.

The RUSP is the list of conditions the HHS secretary recommends for universal newborn screening. States choose whether to adopt each screening, and these decisions can reshape health outcomes for millions of families. HHS’ Health Resources and Services Administration (HRSA) administers the RUSP.

“Early data changes the entire trajectory for affected children and their families,” said HRSA Administrator Tom Engels. “A confirmed result in infancy replaces years of guesswork and helps families make informed decisions about treatment and support.”

Several Members of Congress alongside patient advocates and their families joined Secretary Kennedy to celebrate today’s action.

“By adding DMD and MLD to the Recommended Uniform Screening Panel, this Administration is taking important steps to give parents and families the most information as early as possible to make potentially lifesaving decisions for their children. I’m thrilled to celebrate this announcement that will give families hope as we continue to fight for our nation’s most vulnerable,” said Senator Katie Britt (R-AL).

“Nearly 25 years ago, a Mississippian visited my office and told me his son had been diagnosed with DMD. That conversation kicked off decades of work. It has been among my greatest joys in Congress to help combat DMD and other muscular dystrophies. We have seen a direct relationship between increased research funding and longer lifespans for children diagnosed with these terrible diseases. We have also learned that earlier testing transforms lives. Including DMD in universal screening for newborns will be transformational. I will continue to bring awareness to DMD. I encourage every state to implement this life-changing screening,” said Senator Roger Wicker (R-MS).

“I championed Right to Try to give those with terminal or rare diseases hope and the freedom to try experimental drugs and therapies. I am grateful to Secretary Kennedy and to all those advocating for early screening of rare diseases to give families the chance to act when treatment can be most effective,” said Senator Ron Johnson (R-WI).

“Today’s announcement represents a monumental breakthrough grounded in human compassion. As Co-Chair of the Congressional Rare Disease Caucus, I have long worked to ensure that no child suffers due to a lack of diagnosis, and expanding newborn screening is a critical part of that effort,” said Congressman Gus Bilirakis (R-FL). “Including conditions like MLD and DMD in the newborn screening process is a major step forward. By identifying rare diseases early, we can dramatically improve a child’s chances for timely treatment, effective therapies, and, in some cases, cures.”

HHS will continue to support states as they move toward implementation and evaluate how early detection can improve the lives of children affected by rare diseases.