IGT Applauds Administration’s Decision to add MLD, DMD to the Recommended Uniform Screening Panel

The Health Resources and Services Administration (HRSA) has announced the decision to add metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) to the federal Recommended Uniform Screening Panel (RUSP). IGT Chairman Congressman Erik Paulsen issued the following statement:

“IGT is grateful for the leadership of U.S. Department of Health and Human Services Secretary Robert F. Kennedy, Jr. and HRSA Administrator Tom Engels in recognizing the urgent need for earlier identification of children with MLD and DMD through newborn screening. This decision marks a critical step toward ensuring that children can benefit from early diagnosis and rapidly advancing treatments for these devastating conditions. As innovative gene therapies and other transformative treatments continue to emerge, it is essential that our newborn screening system keep pace so that families are not left waiting for a diagnosis while irreversible damage occurs.

“By adding MLD and DMD to the RUSP, federal leaders are helping to ensure that more children can be connected to appropriate care, clinical trials, and potential gene therapies at the time when these interventions can have the greatest impact. IGT thanks Congressional leaders Senator Katie Britt (R-AL), Senator Roger Wicker (R-MS), Senator Ron Johnson (R-WI) and Congressman Gus Bilirakis (R-FL) for their recognition of the significance of newborn screening and its value for efforts to improve health care and outcomes in the U.S.

“IGT is dedicated to working with federal and state partners to support timely implementation of these additions to the RUSP and to advance broader newborn screening reforms that fully realize the promise of innovation for patients and families affected by serious genetic diseases.​”