In this video from Neurology Today, Dr. Brent Fogel, an associate professor of neurology and human genetics at the University of California, Los Angeles discusses two different types of treatment for spinal muscular atrophy (SMA).
Dr. Fogel talks about the effectiveness of the newly FDA-approved drug nusinersen (Spinraza) and gene therapy agent AVXS-101. He explains that nusinersen targets the SMN2 gene, allowing the splicing pattern to be changed, producing more of the SMN1 protein. The clinical trials of the drug showed that many infants with SMA type 1 reached many milestones not usually reached. However, it’s still unclear how long SMA patients would need to continue to have nusinersen therapy for it to remain effective.
AVXS-101 is a gene therapy which is currently being trialed. It is delivered to the patient through a virus that affects the SMN cells and delivers the gene.
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