In SMA, individuals lack a working copy of the SMN1 gene, and the ASOs are used to facilitate proper splicing of the SMN2 gene through inducing the inclusion of exon 7ultimately rescuing the expression of the SMN protein within in vitro mammalian cell models [91] In 2016, the FDA authorized the use of Spinraza, the first drug-based therapy for SMA [93]
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Similarly, environmental enrichment, which provides enhanced sensory, cognitive, and motor stimulation, can elevate BDNF levels and, when combined with therapies that bolster TrkB expression (as demonstrated with the peptide P42 in an HD mouse model), synergistically delays disease progression and improves motor and cognitive outcomes (Couly et al., 2021)
The move comes as his administration seeks to impose tariffs on pharmaceutical imports that many experts have said could lead to drug shortages and price hikes