Japan’s cost-effectiveness assessment (CEA) panel has approved a revised handling policy for the CEA system for FY2026, with the health…
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Impact of Oligonucleotides - 2: Expert Says Spinraza Changed SMA Treatment Paradigm, Has High Expectation for Zolgensma Too
“Without an effective treatment, 95% of children with spinal muscular atrophy (SMA) require a tracheotomy by their second birthday, but babies who received Spinraza (nusinersen) in clinical trials were able to sing well and speak after the age of three.”…
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