Health and Human Services Secretary Robert F. Kennedy Jr. has focused on reducing health burdens among children, but his department recently eliminated an advisory committee on genetic disorders in newborns and kids. This committee played a central role in making recommendations on which conditions to include in newborn screening, identifying around 14,000 babies per year with potentially life-threatening conditions. The elimination of the committee has raised concerns about the future of newborn screenings for rare disorders like metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD).
Without the committee’s guidance, there is little chance these conditions will be added to the universal screening panel. Only a few states currently screen newborns for MLD or DMD, and advocates fear a return to a patchwork system where some states offer comprehensive screenings while others do not. Advocates are urging Kennedy to reinstate the advisory committee to ensure evidence-based recommendations are made about which conditions to screen for.
Early diagnosis and treatment can significantly impact the quality of life for children with rare genetic disorders like MLD and DMD. Patients can benefit from treatments like gene therapy, which may extend their lives or improve their overall well-being. The elimination of the advisory committee has sparked concern among doctors and advocates about the future of newborn screening and the importance of having an independent body make recommendations based on evidence, not just advocacy.
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