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

On 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.

Headlines

Government Officials:

Advocates & Organizations:

Medical and Scientific Professionals:

Headlines:

• Reuters: Kennedy approves adding two rare disorders to newborn screenings
• Newsweek: HHS Official - Newborn Screening Saves Lives and Empowers Families | Opinion
• Epoch Times: Health Department Recommends 2 New Conditions for Newborn Screening
• Newsmax Health: Kennedy Adds Two Rare Disorder Newborn Screenings
• Seeking Alpha: Sarepta rises as HHS adds Duchenne muscular dystrophy to newborn screenings
• STAT: Two rare diseases added to newborn screening recommendations
• Just the News: RFK Jr. adds fast-moving, debilitating disorders to newborn screening recommendations
• The Baltimore Banner: Metachromatic leukodystrophy and Duchenne’s added to federal newborn screening recommendations
• Precision Medicine Online: HHS Adds Two Rare Genetic Disorders to Newborn Screening Recommendations
• MLive: 2 rare diseases added to newborn screening guidelines
• Meyka: Secretary Kennedy Boosts Rare Disease Detection: What It Means for Bio

Government Officials:

• Sen. Susan Collins via X: “It is wonderful news that the Department of Health and Human Services has announced its decision to add Duchenne muscular dystrophy (DMD) to the Recommended Uniform Screening Panel for newborn children, following a written request from me and a bipartisan group of nine congressional colleagues. Throughout my Senate service, supporting research and early detection for Duchenne muscular dystrophy and other rare diseases has been one of my highest priorities. DMD is often not diagnosed until patients are four or five years old, when meaningful muscle loss has already occurred. This decision from HHS will allow families to get answers at birth and take advantage of the growing list of FDA-approved therapies at a time when they can make the greatest difference in slowing the progression of this devastating disease.”
• Sen. Ron Johnson via X: “I championed Right to Try to give those with terminal or rare diseases hope and the freedom to try experimental drugs and therapies. Thank you @SecKennedy and to all those advocating for early screening of rare diseases to give families the chance to act when treatment can be most effective.”
• Sen. Ron Johnson via Newsmax: “Well, I'm a big supporter of Secretary Kennedy. This is just one example - he has expanded the recommended screening of two rare diseases Duchenne muscular dystrophy… So if you get screened, you can do treatment that is actually effective early on in case instead of waiting five, six, seven years. This is incredibly important. But it's Secretary Kennedy, it's his leadership oftentimes pushing back against the entrenched bureaucracy within the health care agencies. Right to try will be a key part of this because early screening sometimes they're not approved drugs. Some of these parents, they want to use their freedom to have the right to try some of these things. These things work in combination with each other.”
• Sen. Katie Britt via X: “Grateful to join @SecKennedy and incredible family advocates for this announcement today.
• Sen. Katie Britt via X: “Honored to stand with Celia Grace and her parents among countless other family advocates during @SecKennedy’s announcement to add DMD and MLD to the Recommended Uniform Screening Panel. This will help states give parents and families the critical testing needed so more children like Celia Grace have the opportunity to live their American Dream.”
• Rep. Gus Bilirakis via X: “Proud to attend today’s compassionate announcement by Secretary Kennedy. As Co-Chair of the Congressional Rare Disease Caucus, I’ve worked to expand newborn screening so no child suffers from a lack of diagnosis. This action will help ensure earlier diagnoses, better treatments, and more hope for families-while advancing research and innovation for rare diseases. Also honored to stand with the Beacham family and their daughter, Mattie, whose story underscores why this issue matters. If Mattie’s rare disease would have been diagnosed at birth she could have avoided much of the pain she has endured in her short life.  I want to ensure children like Mattie have the best possible start in life.”
• Rep. Gus Bilirakis via X: “Proud to stand with the Beacham family and their daughter Mattie as newborn screening is expanded to include more rare diseases. If Mattie had been diagnosed at birth, she could have avoided much of the pain she’s endured. Thank you to @SecKennedy for leading with compassion-early screening means earlier care, more hope, and a better start for every child.”
• Rep. Buddy Carter via X: “Thanks to the leadership of @SecKennedy, the U.S. Department of Health and Human Services now recommends screening newborns for Duchenne Muscular Dystrophy (DMD) and Metachromatic Leukodystrophy (MLD). Early detection of MLD & DMD will save lives by enabling timely access to care.”
• Rep. Robert Aderholt via X: “Today’s announcement from @SecKennedy and HHS ensures families have the tools for a brighter future and a better quality of life. Thank you, Secretary Kennedy, for your leadership in putting this plan into action.”

Advocates & Organizations:

• Parent Project Muscular Dystrophy and Muscular Dystrophy Association: “Adding Duchenne to the RUSP greatly accelerates the timeframe in which newborns across the country will be screened, allowing families to connect with care and neuromuscular experts sooner and access approved therapies and supportive interventions earlier. By reducing the diagnostic delays that have affected Duchenne families for generations, early identification will lead to improved long-term health outcomes.”
• National Organization for Rare Disorders (@RareDiseases): “Exciting news for the #Duchenne #MuscularDystrophy and #MLD communities, and everyone committed to robust #NewbornScreening across America. NORD applauds #HHS for adding these two rare disorders to the #RUSP so babies with these conditions receive earlier diagnoses and life-saving medical interventions. Read more: https://rarediseases.org/nord-ceo-statement-on-the-addition-of-metachromatic-leukodystrophy-mld-and-duchenne-muscular-dystrophy-dmd-to-the-recommended-uniform-screening-panel/”
• Pamela Gavin, Chief Executive Officer of the National Organization for Rare Disorders: “NORD is grateful for Secretary Kennedy’s decision to add MLD and DMD to the recommended panel, a decision that marks an important step toward universal screening for these two conditions across the country.”
• Pat Furlong, founder of Parent Project Muscular Dystrophy: “Thrilled for this day…A long time coming with a few bumps. But today, that diagnostic odyssey ends.”
• Paul Melmeyer, MPP, Executive Vice President, Public Policy and Advocacy at the Muscular Dystrophy Association: “This achievement reflects the dedication of hundreds of families, dozens of clinicians and researchers, and many partners who have championed timely detection for years…It is a testament to the power of collaboration and this community's shared commitment to ensuring every child with Duchenne has the best possible start in life.”
• Kendra Riley, a Phoenix-area mother and MLD advocate: “Newborn screening for MLD is so important because if a child is not diagnosed prior to the onset of symptoms, they may become ineligible for treatment, as was the case for my middle daughter Livvy, who is currently in hospice dealing with the later stages of this cruel disease… Our family’s story is far too common today, but as newborn screening for MLD becomes more widely available, future generations could be spared from having to watch one child slip away in order to save another. The combination of newborn screening to diagnose MLD at birth and treatment availability changes the trajectory of this disease for children and families.”
• Kendra Riley, a Phoenix-area mother and MLD advocate: “I’m ecstatic that we’re changing the world for other families…”
• Susan Sullivan, a Maryland mother whose son died of MLD in 2018: “…I’m happy to know that children now and in the future will not have to undergo the devastating disease that he did.”
• Jenn McNary @jennmcnary: “Deeply grateful for @HHSGov @RobertKennedyJr in adding #NBS for duchenne and MLD. In further commitment to rare disease patients and a comfort to families, Secretary Kennedy committed that young men and boys like my son max will not lose access to approved exon skipping therapies like exondys, amondys, vyondys and viltepso.   Thank you!!!!”
• MAHA Action (@MAHA_Action): “RFK Jr. has announced that newborn screenings will now check babies for rare diseases. “Children deserve better especially because the tools to improve their health outcomes already exist.” “Newborn screening isn’t just a medical advancement, but a moral imperative.” “With current levels of screening most MLD and DMD diagnosis are too late.” “This is a major reason why approximately 30% of the children with rare diseases do not make it to age five.” “Timely care is compassionate care, and our children deserve nothing less.”
• MAHA Action (@MAHA_Action): “As my uncle, President John F. Kennedy once said, ‘Although children may be the victims of fate, they will not be the victims of our neglect.’” RFK Jr. says HHS will no longer ignore rare childhood diseases like DMD and MLD that have been devastating the health of many American children. “One in 40,000 newborns are born with MLD and DMD affects one in every 5,000 newborn males.” “These so-called rare diseases are not actually that rare.”
• EveryLife Foundation @EveryLifeOrg: “Thank you to Secretary @RobertKennedyJr and @HHSGov for demonstrating a strong commitment to newborn screening and the federal RUSP.”

Medical and Scientific Professionals:

• Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics: “Newborn screening will prove critical as it is the only practical means of diagnosing patients prior to the onset of symptoms, which is key to achieving optimal and equitable outcomes for children in the U.S. and their families dealing with this rapidly progressive, irreversible and ultimately fatal disease…We commend the U.S. Health and Human Services leadership for recognizing the immense and urgent medical need to screen for MLD at birth.”
• Barbara Burton, M.D., attending physician, genetics, genomics and metabolism at the Edwards Family Division of Genetics and Rare Diseases at the Ann & Robert H. Lurie Children’s Hospital of Chicago: “As with many rare, life-threatening diseases, early detection and diagnosis is key to ensuring the best possible outcomes for patients…I have seen first-hand the disparity of outcomes for treated versus untreated children, and I believe we are obligated to provide those with MLD the best opportunity for a meaningful life, which is only possible with universal newborn screening.”
• Michael Gelb, Ph.D., chair in the Departments of Chemistry and Biochemistry at the University of Washington: “Having been passionately involved in the advancement of newborn screening for many severe genetic diseases, I can confidently say few conditions so obviously meet widely accepted inclusion criteria in national programs than MLD…”
• Bo Cumbo: “Solid Biosciences President and CEO Bo Cumbo in a statement told Precision Medicine Online that the company was "thrilled" to see DMD added to the RUSP. "Earlier detection means earlier access to specialized care and innovative therapies, which may potentially have a profound impact on the trajectory for those living with Duchenne," he said.”
• Solid Biosciences: “Today marks a significant milestone for the Duchenne muscular dystrophy community. The U.S. Department of Health and Human Services (HHS) has officially added Duchenne to the Recommended Uniform Screening Panel (RUSP) - the list of conditions recommended for universal newborn screening across the United States. Solid Biosciences is proud to be a longtime supporter of @ParentProjectMD’s efforts to add Duchenne to the RUSP. Earlier detection can lead to earlier care, more informed decision making, and potentially improved outcomes for children and families living with Duchenne. We are thrilled to see newborn screening become a reality for the Duchenne community. Learn more: https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-announces-duchenne-muscular-dystrophy-added https://hhs.gov/press-room/secretary-kennedy-adds-duchenne-muscular-dystrophy-metachromatic-leukodystrophy-to-newborn-screenings.html #Duchenne #DuchenneMuscularDystrophy #NewbornScreening”
• Sarepta: “Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, applauds the addition of Duchenne muscular dystrophy (Duchenne) to the U.S. Recommended Uniform Screening Panel (RUSP). The inclusion of Duchenne on the RUSP represents a pivotal advancement for the Duchenne community, encouraging broader newborn screening at the state level and empowering more families with early diagnosis and timely information to pursue earlier care, including access to available therapies and clinical trials.”
• Diane Berry, Ph.D., executive vice president and chief global policy & advocacy officer at Sarepta: “We commend the Department of Health and Human Services for recognizing the urgent need for early identification and taking action to change the trajectory of this disease.”