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April 27, 2020

Affording Genetic Therapies in the Medicaid Program

Author Affiliations
  • 1Department of Health Policy & Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland
  • 2Johns Hopkins Drug Access and Affordability Initiative, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland
JAMA Pediatr. 2020;174(6):523-524. doi:10.1001/jamapediatrics.2020.0168

Therapies that insert genes, inactivate malfunctioning genes, or alter gene expression have marked therapeutic potential for diseases of childhood. Two recently approved treatments for infantile-onset spinal muscular atrophy type 1 (SMA), which was previously uniformly fatal in early childhood, can increase survival among children. Potentially transformative genetic therapies are in the pipeline for Duchenne muscular dystrophy, sickle cell disease, and cystic fibrosis, among other conditions.

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