Evolving Treatment Options for Pediatric Spinal Muscular Atrophy

Nancy L. Kuntz, MD


July 29, 2020

Until recently, treatment for spinal muscular atrophy (SMA) relied exclusively on supportive therapy, including proper nutrition, ventilatory support, and attention to metabolic bone health.

Because researchers have identified the specific mutation in the SMN1 gene that causes SMA, life-changing treatment options are now available for very young children and infants.

Dr Nancy Kuntz, of the Feinberg School of Medicine at Northwestern University, highlights two groundbreaking, FDA-approved therapies: nusinersen, an antisense oligonucleotide; and onasemnogene abeparvovec-xioi, a gene-replacement therapy.


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