On December 16, 2025, the U.S. Department of Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr. approved the addition of Duchenne Muscular Dystrophy (DMD) and Metachromatic Leukodystrophy (MLD) to the Recommended Uniform Screening Panel (RUSP). This decision follows a scientific review process and public comment period. The move aims to enable early detection of both conditions so that children can receive FDA-approved therapies at the most effective time, potentially slowing disease progression and improving quality of life.
The decision has drawn responses from lawmakers, advocacy groups, families, and medical professionals. Senator Susan Collins said, “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.”
Senator Ron Johnson commented on social media: “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.” In an interview with Newsmax he added: “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.”
Senator Katie Britt also voiced her support: “Grateful to join @SecKennedy and incredible family advocates for this announcement today." She continued: "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.”
Congressman Gus Bilirakis stated: “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.” He added: “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.”
Other members such as Representative Buddy Carter noted: “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.” Representative Robert Aderholt remarked: “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.”
Advocacy organizations also responded positively:
Parent Project Muscular Dystrophy together with Muscular Dystrophy Association stated: “Adding Duchenne to the RUSP greatly accelerates the timeframe in which newborns across the country will be screened allowing families connect with care… By reducing diagnostic delays that have affected Duchenne families for generations early identification will lead improved long-term health outcomes.”
The National Organization for Rare Disorders said via social media: “Exciting news for #Duchenne #MuscularDystrophy & #MLD communities… NORD applauds #HHS adding these two rare disorders #RUSP so babies… receive earlier diagnoses & life-saving interventions.” Pamela Gavin CEO at NORD commented further: “NORD is grateful for Secretary Kennedy’s decision… marks an important step toward universal screening…”
Pat Furlong founder Parent Project Muscular Dystrophy expressed appreciation saying: "Thrilled...that diagnostic odyssey ends."
Paul Melmeyer Executive Vice President Public Policy Advocacy Muscular Dystrophy Association stated: "This achievement reflects dedication hundreds families... clinicians researchers... testifies power collaboration..."
Kendra Riley Phoenix-area mother MLD advocate shared her experience about delayed diagnosis impacting treatment eligibility stating that widespread newborn screening could spare future generations similar hardship.
Medical professionals echoed support:
Bobby Gaspar MD PhD CEO Orchard Therapeutics said: "Newborn screening... only practical means diagnosing patients prior onset symptoms... commend US Health Human Services recognizing immense urgent medical need screen MLD at birth."
Barbara Burton MD attending physician Ann & Robert H Lurie Children’s Hospital Chicago noted importance universal access saying disparities exist between treated untreated children.
Michael Gelb PhD University Washington emphasized few conditions meet inclusion criteria national programs as clearly as MLD.
Bo Cumbo President CEO Solid Biosciences told Precision Medicine Online earlier detection allows access specialized care innovative therapies potentially affecting disease trajectory.
Sarepta Therapeutics issued statement calling addition pivotal advancement encouraging broader state-level implementation empowering more families pursue earlier care including clinical trials; Diane Berry Ph.D., EVP Chief Global Policy Advocacy Officer Sarepta commended HHS's recognition urgent need early identification.
News coverage reflected broad interest in these developments across outlets including Reuters Newsweek STAT Epoch Times Newsmax Precision Medicine Online The Baltimore Banner Just The News among others.
