Health Secretary Robert F. Kennedy Jr. approved two new screenings for newborns, adding Duchenne muscular dystrophy and Metachromatic leukodystrophy, or DMD and MLD, respectively, to the list of recommended screenings.
“As my uncle has said, ‘although children may be the victims of fate, they will not be the victims of our neglect,’” Kennedy said in a statement. “This action honors that principle. Early screening gives every child a fair chance at timely, effective care, and it delivers families the answers and treatment options they deserve—right when they need them most.”
Children with the disorders are often diagnosed at ages four or five, following significant muscle loss or functional decline. States can choose whether to adopt the screenings.
“Not only will adding these conditions to the RUSP help children retain abilities for a longer period, but it also gives families a better chance to avoid the long delays, repeated specialist visits, and financial and emotional strain that often define the years-long diagnostic search for rare diseases,” an HHS press release explains.
Tom Engels, the administrator of the department’s Health Resources and Services Administration, said, “Early data changes the entire trajectory for affected children and their families. A confirmed result in infancy replaces years of guesswork and helps families make informed decisions about treatment and support.”
According to the CDC, other conditions newborns may be screened for include “spinal muscular atrophy, cystic fibrosis, sickle cell disease and other hemoglobinopathies, endocrine diseases, inborn errors of metabolism, lysosomal storage diseases, severe combined immunodeficiencies, critical congential heart defects, and hearing loss.”
