HHS Takes a Lifesaving Step Forward for Newborns in Tribal Communities

When it comes to children born with rare diseases, early diagnosis can make the difference between life and death. Since many rare diseases come with a short treatment window – usually before symptoms appear in a child’s first months or years – early screening and diagnosis is essential for ensuring children have the best possible chance at a healthy, flourishing life.

Last month, Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr. made a historic announcement that will significantly improve outcomes for newborns across the country, especially in tribal communities: the addition of Duchenne Muscular Dystrophy (DMD) and Metachromatic Leukodystrophy (MLD) to the Recommended Uniform Screening Panel (RUSP).

The RUSP is the national guideline for newborn genetic disease screening that states and use to inform decisions about their respective panels. When conditions are added to the RUSP, states and federally funded health programs are better positioned to screen infants early and help families overcome barriers to care before their child’s effective treatment window runs out.

The addition of these rare degenerative diseases to the RUSP will encourage early screening and diagnosis, making treatment possible for newborns who otherwise would have no hope of recovery. By expanding the RUSP, HHS is helping reduce barriers to screening and treatment or all Americans, including American Indian and Alaska Native communities where access to specialty care can be limited.

“Every child deserves timely, compassionate care and a fair chance to overcome rare disease—including children in tribal communities, who face higher rates of devastating conditions like MLD,” said Secretary Kennedy. “This action strengthens access to care, supports families, and will save lives across Indian Country.” 

For the Navajo Nation, this announcement carries particular weight. Research conducted in the western portion of the Nation has found that MLD occurs in approximately 1 in every 2,520 live births, with an estimated carrier rate between 1 in 25 and 1 in 50—far higher than in the general population. MLD is a rapidly progressive neurodegenerative disease that affects children younger than five. Without detection and treatment before symptoms appear, it is uniformly fatal. These additions help ensure that all children, including Navajo newborns who face higher rates of MLD, have access to timely diagnoses and treatments such as emerging gene therapies that must be given early to be effective.

“Secretary Kennedy’s leadership in rare disease screening and research is an important step in addressing the health challenges facing American Indian and Alaska Native communities,” said Mark Cruz, senior advisor to Secretary Kennedy for tribal affairs. “This action assures parents that their children will receive needed attention. Screening at birth will prevent many late MLD diagnoses and give Native children the chance to grow up healthy.” 

The addition of DMD and MLD to the RUSP marks more than a policy update -- it is a promise to the families impacted by rare disease that they are not alone and that hope, treatment, and flourishing lives are possible for their children. By expanding pathways to rare disease screening and treatment, HHS is keeping that promise to families across America’s states and tribal communities.

“I am deeply grateful for Secretary Kennedy’s leadership and his continued commitment to advancing the health and well-being of the Navajo people and all of Indian Country,” said Navajo Nation President Buu Nygren. “His dedication to helping our communities heal and thrive reflects the kind of partnership we need to build a stronger, healthier future for Native people everywhere. This effort represents an important step in the right direction, one rooted in compassion, understanding, and respect for our shared values. We appreciate Secretary Kennedy’s vision and his steadfast commitment to making Native America healthy again.”  

With continued partnership between tribal leaders and Indian Health Service programs, HHS’ historic rare disease detection initiative will change the lives of countless families and children across Indian Country.