First Comprehensive Review of SMA Drugs Offers Long-Term Insights, Outcome Gaps
A review of 21 studies on SMA therapies (Spinraza, Evrysdi, Zolgensma) found motor function improvement in 1,374 patients over 48 months, with early intervention yielding better outcomes. Safety profiles were favorable, but no significant respiratory or nutritional improvements were noted. The high cost and uncertain long-term benefits pose challenges for coverage decisions.
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A review of 21 studies on SMA therapies (Spinraza, Evrysdi, Zolgensma) found motor function improvement in 1,374 patients over 48 months, with early intervention yielding better outcomes. Safety profiles were favorable, but no significant respiratory or nutritional improvements were noted. The high cost and uncertain long-term benefits pose challenges for coverage decisions.