How Spinraza (nusinersen) and Evrysdi treat Spinal Muscular Atrophy (SMA) through splice-switching
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YouTubethe bumbling biochemist
How Spinraza (nusinersen) and Evrysdi treat Spinal Muscular Atrophy (SMA) through splice-switching
Nusinersen (trade name “Spinraza”) has really done wonders for patients with the debilitating genetic neuromuscular disorder Spinal Muscular Atrophy (SMA), the most common genetic cause of infant death in the US. Nusinersen isn’t your typical type of drug - it isn’t “just” a “small molecule” compound like a little chemical ...
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