Panelists discuss how intrathecal delivery of onasemnogene abeparvovec in the STEER study demonstrates statistically significant motor function improvements in older patients with SMA (ages 2-18 years ...
The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in adults and pediatric patients 2 years and older with a confirmed mutation in ...
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