Managed Healthcare Executive August 6, 2024
The advent of gene therapies for spinal muscular atrophy (SMA) has allowed some patients reach developmental milestones when treated as newborns, but the long-term durability of these treatments is unknown. Researchers recently tested a new approach in mice that combines both gene supplementation and genome editing to increase the likelihood of medical intervention lasting longer.
SMA is caused by mutations in the SMN1 gene, leading to the degeneration of motor neurons, muscle weakness, and atrophy. Disease-modifying medications have transformed the treatment landscape, with the availability of Spinraza (nusinersen) and Evrysdi (risdiplam), which don’t cure the SMA but do modify the underlying disease process; and Zolgensma (onasemnogene abeparvovec-xioi), which is a one-time gene replacement therapy.
Zolgensma, approved by the FDA in...
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