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Roche’s Evrysdi significantly improves lives of babies with SMA
The Phase II study of Evrysdi showed significant improvements in survival and motor milestones in babies with type 1 spinal muscular atrophy (SMA).
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Spinal Muscular Atrophy
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Ten drugs in pre-registration likely to be blockbusters by 2025, finds report
New research has found that of the drugs in the pre-registration phase, 10 are expected to become blockbusters over the next six years.
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Novartis could face legal action over Zolgensma application
The pharmaceutical company failed to inform the FDA of manipulated data when the gene therapy was under review and could face a legal battle.
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Late-stage pipeline drugs could compete with only treatment for SMA
A number of drugs currently in their late stages in the development process could see competition for nusinersen, the only current SMA treatment, rise...
