Spinal Muscular Atrophy (SMA)
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Spinal Muscular Atrophy (SMA)
SMA represents one of the greatest success stories in neurology:
- Nusinersen (Spinraza®) – antisense therapy
- Onasemnogene abeparvovec (Zolgensma®) – gene replacement therapy
- Risdiplam (Evrysdi®) – oral SMN2 splicing modifier
Early treatment has transformed SMA from a fatal disease to a manageable condition.
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